Somatic Cell Genome Editing (SCGE)
Somatic Cell Genome Editing (SCGE)
Program Publications
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The search results on this publication page are automated on a monthly schedule based on acknowledgement of NIH Common Fund award numbers and intramural awards. Therefore, this list is not an exhaustive or error-free account of the program’s publications.
| Title | Authors | Journal | PubMedID | Publication Date |
|---|---|---|---|---|
| Engineering a targeted and safe bone anabolic gene therapy to treat osteoporosis in alveolar bone loss. | Lin, Chujiao; Yang, Yeon-Suk; Ma, Hong; Chen, Zhihao; Chen, Dong; John, Aijaz Ahmad; Xie, Jun; Gao, Guangping; Shim, Jae-Hyuck | Molecular therapy : the journal of the American Society of Gene Therapy | 38937970 | 2024 Sep 04 |
| Enhancing in vivo cell and tissue targeting by modulation of polymer nanoparticles and macrophage decoys. | Piotrowski-Daspit, Alexandra S; Bracaglia, Laura G; Eaton, David A; Richfield, Owen; Binns, Thomas C; Albert, Claire; Gould, Jared; Mortlock, Ryland D; Egan, Marie E; Pober, Jordan S; Saltzman, W Mark | Nature communications | 38762483 | 2024 May 18 |
| Rapid measurement of hemoglobin-oxygen dissociation by leveraging Bohr effect and Soret band bathochromic shift. | Sekyonda, Zoe; An, Ran; Goreke, Utku; Man, Yuncheng; Monchamp, Karamoja; Bode, Allison; Zhang, Qiaochu; El-Gammal, Yasmin; Kityo, Cissy; Kalfa, Theodosia A; Akkus, Ozan; Gurkan, Umut A | The Analyst | 38501195 | 2024 Apr 29 |
| A base editing strategy using mRNA-LNPs for in vivo correction of the most frequent phenylketonuria variant. | Brooks, Dominique L; Whittaker, Madelynn N; Said, Hooda; Dwivedi, Garima; Qu, Ping; Musunuru, Kiran; Ahrens-Nicklas, Rebecca C; Alameh, Mohamad-Gabriel; Wang, Xiao | HGG advances | 37922902 | 2024 Jan 11 |
| Combinatorial development of nebulized mRNA delivery formulations for the lungs. | Jiang, Allen Y; Witten, Jacob; Raji, Idris O; Eweje, Feyisayo; MacIsaac, Corina; Meng, Sabrina; Oladimeji, Favour A; Hu, Yizong; Manan, Rajith S; Langer, Robert; Anderson, Daniel G | Nature nanotechnology | 37985700 | 2024 Mar |
| Efficient prime editing in mouse brain, liver and heart with dual AAVs. | Davis, Jessie R; Banskota, Samagya; Levy, Jonathan M; Newby, Gregory A; Wang, Xiao; Anzalone, Andrew V; Nelson, Andrew T; Chen, Peter J; Hennes, Andrew D; An, Meirui; Roh, Heejin; Randolph, Peyton B; Musunuru, Kiran; Liu, David R | Nature biotechnology | 37142705 | 2024 Feb |
| Phage-assisted evolution of highly active cytosine base editors with enhanced selectivity and minimal sequence context preference. | Zhang, Emily; Neugebauer, Monica E; Krasnow, Nicholas A; Liu, David R | Nature communications | 38402281 | 2024 Feb 24 |
| Compartmentalized ocular lymphatic system mediates eye-brain immunity. | Yin, Xiangyun; Zhang, Sophia; Lee, Ju Hyun; Dong, Huiping; Mourgkos, George; Terwilliger, Gordon; Kraus, Aurora; Geraldo, Luiz Henrique; Poulet, Mathilde; Fischer, Suzanne; Zhou, Ting; Mohammed, Farrah Shalima; Zhou, Jiangbing; Wang, Yongfu; Malloy, Seth; Rohner, Nicolas; Sharma, Lokesh; Salinas, Irene; Eichmann, Anne; Thomas, Jean-Leon; Saltzman, W Mark; Huttner, Anita; Zeiss, Caroline; Ring, Aaron; Iwasaki, Akiko; Song, Eric | Nature | 38418880 | 2024 Apr |
| Self-delivering, chemically modified CRISPR RNAs for AAV co-delivery and genome editing in vivo. | Zhang, Han; Kelly, Karen; Lee, Jonathan; Echeverria, Dimas; Cooper, David; Panwala, Rebecca; Amrani, Nadia; Chen, Zexiang; Gaston, Nicholas; Wagh, Atish; Newby, Gregory A; Xie, Jun; Liu, David R; Gao, Guangping; Wolfe, Scot A; Khvorova, Anastasia; Watts, Jonathan K; Sontheimer, Erik J | Nucleic acids research | 38033325 | 2024 Jan 25 |
| Engineered virus-like particles for transient delivery of prime editor ribonucleoprotein complexes in vivo. | An, Meirui; Raguram, Aditya; Du, Samuel W; Banskota, Samagya; Davis, Jessie R; Newby, Gregory A; Chen, Paul Z; Palczewski, Krzysztof; Liu, David R | Nature biotechnology | 38191664 | 2024 Jan 08 |
| Cationic cholesterol-dependent LNP delivery to lung stem cells, the liver, and heart. | Radmand, Afsane; Kim, Hyejin; Beyersdorf, Jared; Dobrowolski, Curtis N; Zenhausern, Ryan; Paunovska, Kalina; Huayamares, Sebastian G; Hua, Xuanwen; Han, Keyi; Loughrey, David; Hatit, Marine Z C; Del Cid, Ada; Ni, Huanzhen; Shajii, Aram; Li, Andrea; Muralidharan, Abinaya; Peck, Hannah E; Tiegreen, Karen E; Jia, Shu; Santangelo, Philip J; Dahlman, James E | Proceedings of the National Academy of Sciences of the United States of America | 38437539 | 2024 Mar 12 |
| A humanized mouse model for adeno-associated viral gene therapy. | Barzi, Mercedes; Chen, Tong; Gonzalez, Trevor J; Pankowicz, Francis P; Oh, Seh Hoon; Streff, Helen L; Rosales, Alan; Ma, Yunhan; Collias, Sabrina; Woodfield, Sarah E; Diehl, Anna Mae; Vasudevan, Sanjeev A; Galvan, Thao N; Goss, John; Gersbach, Charles A; Bissig-Choisat, Beatrice; Asokan, Aravind; Bissig, Karl-Dimiter | Nature communications | 38438373 | 2024 Mar 04 |
| AAV-based gene editing of type 1 collagen mutation to treat osteogenesis imperfecta. | Yang, Yeon-Suk; Sato, Tadatoshi; Chaugule, Sachin; Ma, Hong; Xie, Jun; Gao, Guangping; Shim, Jae-Hyuck | Molecular therapy. Nucleic acids | 38261950 | 2024 Mar 12 |
| Advancements in therapeutic development: kidney organoids and organs on a chip. | Tabibzadeh, Nahid; Morizane, Ryuji | Kidney international | 38296026 | 2024 Apr |
| RNA language models predict mutations that improve RNA function. | Shulgina, Yekaterina; Trinidad, Marena I; Langeberg, Conner J; Nisonoff, Hunter; Chithrananda, Seyone; Skopintsev, Petr; Nissley, Amos J; Patel, Jaymin; Boger, Ron S; Shi, Honglue; Yoon, Peter H; Doherty, Erin E; Pande, Tara; Iyer, Aditya M; Doudna, Jennifer A; Cate, Jamie H D | bioRxiv : the preprint server for biology | 38617247 | 2024 Sep 16 |
| Imaging of adeno-associated viral capsids for purposes of gene editing using CEST NMR/MRI. | Lam, Bonnie; Velasquez, Mark; Ogiyama, Tomoko; Godines, Kevin; Szu, Fan-Yun; Velasquez-Mao, A J; AlGhuraibawi, Wissam; Wang, Jingshen; Messersmith, Phillip B; Vandsburger, Moriel H | Magnetic resonance in medicine | 38651648 | 2024 Aug |
| Imaging CAR-NK cells targeted to HER2 ovarian cancer with human sodium-iodide symporter-based positron emission tomography. | Shalaby, Nourhan; Xia, Ying; Kelly, John J; Sanchez-Pupo, Rafael; Martinez, Francisco; Fox, Matthew S; Thiessen, Jonathan D; Hicks, Justin W; Scholl, Timothy J; Ronald, John A | European journal of nuclear medicine and molecular imaging | 38722382 | 2024 Sep |
| AAV5 Delivery of CRISPR/Cas9 Mediates Genome Editing in the Lungs of Young Rhesus Monkeys. | Liang, Shun-Qing; Navia, Andrew W; Ramseier, Michelle; Zhou, Xuntao; Martinez, Michele; Lee, Charles; Zhou, Chen; Wu, Joae; Xie, Jun; Su, Qin; Wang, Dan; Flotte, Terence R; Anderson, Daniel G; Tarantal, Alice F; Shalek, Alex K; Gao, Guangping; Xue, Wen | Human gene therapy | 38767512 | 2024 Jul 03 |
| Birth of protein folds and functions in the virome. | Nomburg, Jason; Doherty, Erin E; Price, Nathan; Bellieny-Rabelo, Daniel; Zhu, Yong K; Doudna, Jennifer A | Nature | 39187718 | 2024 Sep |
| Neuronal DNA repair reveals strategies to influence CRISPR editing outcomes. | Ramadoss, Gokul N; Namaganda, Samali J; Hamilton, Jennifer R; Sharma, Rohit; Chow, Karena G; Macklin, Bria L; Sun, Mengyuan; Liu, Jia-Cheng; Fellmann, Christof; Watry, Hannah L; Jin, Julianne; Perez, Barbara S; Sandoval Espinoza, Cindy R; Matia, Madeline P; Lu, Serena H; Judge, Luke M; Nussenzweig, Andre; Adamson, Britt; Murthy, Niren; Doudna, Jennifer A; Kampmann, Martin; Conklin, Bruce R | bioRxiv : the preprint server for biology | 38979269 | 2024 Jun 26 |
| Targeted genome editing restores auditory function in adult mice with progressive hearing loss caused by a human microRNA mutation. | Zhu, Wenliang; Du, Wan; Rameshbabu, Arun Prabhu; Armstrong, Ariel Miura; Silver, Stewart; Kim, Yehree; Wei, Wei; Shu, Yilai; Liu, Xuezhong; Lewis, Morag A; Steel, Karen P; Chen, Zheng-Yi | Science translational medicine | 38985856 | 2024 Jul 10 |
| The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV2. | Cui, Mengtian; Su, Qin; Yip, Mitchell; McGowan, Jackson; Punzo, Claudio; Gao, Guangping; Tai, Phillip W L | Gene therapy | 39134629 | 2024 Aug 12 |
| Liver directed adeno-associated viral vectors to treat metabolic disease. | Chuecos, Marcel A; Lagor, William R | Journal of inherited metabolic disease | 37254440 | 2024 Jan |
| Protein-based nanoparticles for therapeutic nucleic acid delivery. | Eweje, Feyisayo; Walsh, Michelle L; Ahmad, Kiran; Ibrahim, Vanessa; Alrefai, Assma; Chen, Jiaxuan; Chaikof, Elliot L | Biomaterials | 38181574 | 2024 Mar |
| CasPEDIA Database: a functional classification system for class 2 CRISPR-Cas enzymes. | Adler, Benjamin A; Trinidad, Marena I; Bellieny-Rabelo, Daniel; Zhang, Elaine; Karp, Hannah M; Skopintsev, Petr; Thornton, Brittney W; Weissman, Rachel F; Yoon, Peter H; Chen, LinXing; Hessler, Tomas; Eggers, Amy R; Colognori, David; Boger, Ron; Doherty, Erin E; Tsuchida, Connor A; Tran, Ryan V; Hofman, Laura; Shi, Honglue; Wasko, Kevin M; Zhou, Zehan; Xia, Chenglong; Al-Shimary, Muntathar J; Patel, Jaymin R; Thomas, Vienna C J X; Pattali, Rithu; Kan, Matthew J; Vardapetyan, Anna; Yang, Alana; Lahiri, Arushi; Maxwell, Micaela F; Murdock, Andrew G; Ramit, Glenn C; Henderson, Hope R; Calvert, Roland W; Bamert, Rebecca S; Knott, Gavin J; Lapinaite, Audrone; Pausch, Patrick; Cofsky, Joshua C; Sontheimer, Erik J; Wiedenheft, Blake; Fineran, Peter C; Brouns, Stan J J; Sashital, Dipali G; Thomas, Brian C; Brown, Christopher T; Goltsman, Daniela S A; Barrangou, Rodolphe; Siksnys, Virginius; Banfield, Jillian F; Savage, David F; Doudna, Jennifer A | Nucleic acids research | 37889041 | 2024 Jan 05 |
| Targeted nonviral delivery of genome editors in vivo. | Tsuchida, Connor A; Wasko, Kevin M; Hamilton, Jennifer R; Doudna, Jennifer A | Proceedings of the National Academy of Sciences of the United States of America | 38437567 | 2024 Mar 12 |
| Building CRISPR Gene Therapies for the Central Nervous System: A Review. | Salomonsson, Sally E; Clelland, Claire D | JAMA neurology | 38285472 | 2024 Mar 01 |
| Recommendations on fit-for-purpose criteria to establish quality management for microphysiological systems and for monitoring their reproducibility. | Pamies, David; Ekert, Jason; Zurich, Marie-Gabrielle; Frey, Olivier; Werner, Sophie; Piergiovanni, Monica; Freedman, Benjamin S; Keong Teo, Adrian Kee; Erfurth, Hendrik; Reyes, Darwin R; Loskill, Peter; Candarlioglu, Pelin; Suter-Dick, Laura; Wang, Shan; Hartung, Thomas; Coecke, Sandra; Stacey, Glyn N; Wagegg, Beren Atac; Dehne, Eva-Maria; Pistollato, Francesca; Leist, Marcel | Stem cell reports | 38670111 | 2024 May 14 |
| Improved specificity and efficacy of base-editing therapies with hybrid guide RNAs. | Whittaker, Madelynn N; Brooks, Dominique L; Quigley, Aidan; Jindal, Ishaan; Said, Hooda; Qu, Ping; Wang, Josh Zhiyong; Ahrens-Nicklas, Rebecca C; Musunuru, Kiran; Alameh, Mohamad-Gabriel; Peranteau, William H; Wang, Xiao | bioRxiv : the preprint server for biology | 38712058 | 2024 Jun 16 |
| Activation of the imprinted Prader-Willi Syndrome locus by CRISPR-based epigenome editing. | Rohm, Dahlia; Black, Joshua B; McCutcheon, Sean R; Barrera, Alejandro; Morone, Daniel J; Nuttle, Xander; de Esch, Celine E; Tai, Derek J C; Talkowski, Michael E; Iglesias, Nahid; Gersbach, Charles A | bioRxiv : the preprint server for biology | 38496583 | 2024 Mar 04 |
| A novel humanized mouse model for HIV and tuberculosis co-infection studies. | Bohórquez, José Alejandro; Adduri, Sitaramaraju; Ansari, Danish; John, Sahana; Florence, Jon; Adejare, Omoyeni; Singh, Gaurav; Konduru, Nagarjun V; Jagannath, Chinnaswamy; Yi, Guohua | Frontiers in immunology | 38799434 | 2024 |
| Advancing preclinical drug evaluation through automated 3D imaging for high-throughput screening with kidney organoids. | Oishi, Haruka; Tabibzadeh, Nahid; Morizane, Ryuji | Biofabrication | 38547531 | 2024 Apr 08 |
| Bioengineered Model of Human LGMD2B Skeletal Muscle Reveals Roles of Intracellular Calcium Overload in Contractile and Metabolic Dysfunction in Dysferlinopathy. | Khodabukus, Alastair; Prabhu, Neel K; Roberts, Taylor; Buldo, Meghan; Detwiler, Amber; Fralish, Zachary D; Kondash, Megan E; Truskey, George A; Koves, Timothy R; Bursac, Nenad | Advanced science (Weinheim, Baden-Wurttemberg, Germany) | 38887849 | 2024 Aug |
| Adeno-associated virus as a delivery vector for gene therapy of human diseases. | Wang, Jiang-Hui; Gessler, Dominic J; Zhan, Wei; Gallagher, Thomas L; Gao, Guangping | Signal transduction and targeted therapy | 38565561 | 2024 Apr 03 |
| Genetics of cystogenesis in base-edited human organoids reveal therapeutic strategies for polycystic kidney disease. | Vishy, Courtney E; Thomas, Chardai; Vincent, Thomas; Crawford, Daniel K; Goddeeris, Matthew M; Freedman, Benjamin S | Cell stem cell | 38579684 | 2024 Apr 04 |
| Bacteriophage therapy for the treatment of Mycobacterium tuberculosis infections in humanized mice. | Yang, Fan; Labani-Motlagh, Alireza; Bohorquez, Jose Alejandro; Moreira, Josimar Dornelas; Ansari, Danish; Patel, Sahil; Spagnolo, Fabrizio; Florence, Jon; Vankayalapati, Abhinav; Sakai, Tsuyoshi; Sato, Osamu; Ikebe, Mitsuo; Vankayalapati, Ramakrishna; Dennehy, John J; Samten, Buka; Yi, Guohua | Communications biology | 38461214 | 2024 Mar 09 |
| Hachiman is a genome integrity sensor. | Tuck, Owen T; Adler, Benjamin A; Armbruster, Emily G; Lahiri, Arushi; Hu, Jason J; Zhou, Julia; Pogliano, Joe; Doudna, Jennifer A | bioRxiv : the preprint server for biology | 38464307 | 2024 Feb 29 |
| Long-term efficacy and safety of cardiac genome editing for catecholaminergic polymorphic ventricular tachycardia. | Moore, Oliver M; Aguilar-Sanchez, Yuriana; Lahiri, Satadru K; Hulsurkar, Mohit M; Alberto Navarro-Garcia, J; Word, Tarah A; Keefe, Joshua A; Barazi, Dean; Munivez, Elda M; Moore, Charles T; Parthasarathy, Vaidya; Davidson, Jaysón; Lagor, William R; Park, So Hyun; Bao, Gang; Miyake, Christina Y; Wehrens, Xander H T | The journal of cardiovascular aging | 38464671 | 2024 Jan |
| Branched chemically modified poly(A) tails enhance the translation capacity of mRNA. | Chen, Hongyu; Liu, Dangliang; Guo, Jianting; Aditham, Abhishek; Zhou, Yiming; Tian, Jiakun; Luo, Shuchen; Ren, Jingyi; Hsu, Alvin; Huang, Jiahao; Kostas, Franklin; Wu, Mingrui; Liu, David R; Wang, Xiao | Nature biotechnology | 38519719 | 2024 Mar 22 |
| Interferon-γ induces combined pyroptotic angiopathy and APOL1 expression in human kidney disease. | Juliar, Benjamin A; Stanaway, Ian B; Sano, Fumika; Fu, Hongxia; Smith, Kelly D; Akilesh, Shreeram; Scales, Suzie J; El Saghir, Jamal; Bhatraju, Pavan K; Liu, Esther; Yang, Johnson; Lin, Jennie; Eddy, Sean; Kretzler, Matthias; Zheng, Ying; Himmelfarb, Jonathan; Harder, Jennifer L; Freedman, Benjamin S | Cell reports | 38838223 | 2024 Jun 25 |
| Efficient site-specific integration of large genes in mammalian cells via continuously evolved recombinases and prime editing. | Pandey, Smriti; Gao, Xin D; Krasnow, Nicholas A; McElroy, Amber; Tao, Y Allen; Duby, Jordyn E; Steinbeck, Benjamin J; McCreary, Julia; Pierce, Sarah E; Tolar, Jakub; Meissner, Torsten B; Chaikof, Elliot L; Osborn, Mark J; Liu, David R | Nature biomedical engineering | 38858586 | 2024 Jun 10 |
| T Cell Responses during Human Immunodeficiency Virus/Mycobacterium tuberculosis Coinfection. | Bohórquez, José Alejandro; Jagannath, Chinnaswamy; Xu, Huanbin; Wang, Xiaolei; Yi, Guohua | Vaccines | 39204027 | 2024 Aug 09 |
| Amphiphilic shuttle peptide delivers base editor ribonucleoprotein to correct the CFTR R553X mutation in well-differentiated airway epithelial cells. | Kulhankova, Katarina; Cheng, Anna X; Traore, Soumba; Auger, Maud; Pelletier, Mia; Hervault, Maxime; Wells, Kevin D; Green, Jonathan A; Byrne, Addison; Nelson, Benjamin; Sponchiado, Mariana; Boosani, Chandra; Heffner, Caleb S; Snow, Kathy J; Murray, Stephen A; Villacreses, Raul A; Rector, Michael V; Gansemer, Nicholas D; Stoltz, David A; Allamargot, Chantal; Couture, Frédéric; Hemez, Colin; Hallée, Stéphanie; Barbeau, Xavier; Harvey, Mario; Lauvaux, Coraline; Gaillet, Bruno; Newby, Gregory A; Liu, David R; McCray Jr, Paul B; Guay, David | Nucleic acids research | 39315713 | 2024 Sep 24 |
| Preexisting maternal immunity to AAV but not Cas9 impairs in utero gene editing in mice. | Riley, John S; Luks, Valerie L; Berkowitz, Cara L; Dumitru, Ana Maria; Kus, Nicole J; Dave, Apeksha; Menon, Pallavi; De Paepe, Monique E; Jain, Rajan; Li, Li; Dugoff, Lorraine; Teefey, Christina Paidas; Alameh, Mohamad-Gabriel; Zoltick, Philip W; Peranteau, William H | The Journal of clinical investigation | 38950310 | 2024 May 09 |
| Cryopreservation of human kidney organoids. | Mashouf, Parham; Tabibzadeh, Nahid; Kuraoka, Shohei; Oishi, Haruka; Morizane, Ryuji | Cellular and molecular life sciences : CMLS | 39023560 | 2024 Jul 18 |
| CRISPR-Cas12a bends DNA to destabilize base pairs during target interrogation. | Soczek, Katarzyna M; Cofsky, Joshua C; Tuck, Owen T; Shi, Honglue; Doudna, Jennifer A | bioRxiv : the preprint server for biology | 39131396 | 2024 Jul 31 |
| Tumor-tropic Trojan horses: Using mesenchymal stem cells as cellular nanotheranostics. | Rosu, Ana; Ghaemi, Behnaz; Bulte, Jeff W M; Shakeri-Zadeh, Ali | Theranostics | 38169524 | 2024 |
| Validated assays for the quantification of C9orf72 human pathology. | Salomonsson, S E; Maltos, A M; Gill, K; Aladesuyi Arogundade, O; Brown, K A; Sachdev, A; Sckaff, M; Lam, K J K; Fisher, I J; Chouhan, R S; Van Laar, V S; Marley, C B; McLaughlin, I; Bankiewicz, K S; Tsai, Y-C; Conklin, B R; Clelland, C D | Scientific reports | 38191789 | 2024 Jan 08 |
| Engineering self-deliverable ribonucleoproteins for genome editing in the brain. | Chen, Kai; Stahl, Elizabeth C; Kang, Min Hyung; Xu, Bryant; Allen, Ryan; Trinidad, Marena; Doudna, Jennifer A | Nature communications | 38409124 | 2024 Feb 26 |
| Mechanosensitive genomic enhancers potentiate the cellular response to matrix stiffness. | Cosgrove, Brian D; Bounds, Lexi R; Taylor, Carson Key; Su, Alan L; Rizzo, Anthony J; Barrera, Alejandro; Crawford, Gregory E; Hoffman, Brenton D; Gersbach, Charles A | bioRxiv : the preprint server for biology | 38260455 | 2024 Jan 10 |
| Harnessing non-Watson-Crick's base pairing to enhance CRISPR effectors cleavage activities and enable gene editing in mammalian cells. | Gao, Shuliang; Guan, Huiwen; Bloomer, Hanan; Wich, Douglas; Song, Donghui; Khirallah, Jennifer; Ye, Zhongfeng; Zhao, Yu; Chen, Mengting; Xu, Chutian; Liu, Lihan; Xu, Qiaobing | Proceedings of the National Academy of Sciences of the United States of America | 38150477 | 2024 Jan 09 |
| Optimization of base editors for the functional correction of SMN2 as a treatment for spinal muscular atrophy. | Alves, Christiano R R; Ha, Leillani L; Yaworski, Rebecca; Sutton, Emma R; Lazzarotto, Cicera R; Christie, Kathleen A; Reilly, Aoife; Beauvais, Ariane; Doll, Roman M; de la Cruz, Demitri; Maguire, Casey A; Swoboda, Kathryn J; Tsai, Shengdar Q; Kothary, Rashmi; Kleinstiver, Benjamin P | Nature biomedical engineering | 38057426 | 2024 Feb |
| TTN truncation variants produce sarcomere-integrating proteins of uncertain functional significance. | Hinson, J Travis; Campbell, Stuart G | The Journal of clinical investigation | 38226618 | 2024 Jan 16 |
| CRISPR Activation Reverses Haploinsufficiency and Functional Deficits Caused by TTN Truncation Variants. | Ghahremani, Shahnaz; Kanwal, Aditya; Pettinato, Anthony; Ladha, Feria; Legere, Nicholas; Thakar, Ketan; Zhu, Yanfen; Tjong, Harianto; Wilderman, Andrea; Stump, W Tom; Greenberg, Lina; Greenberg, Michael J; Cotney, Justin; Wei, Chia-Lin; Hinson, J Travis | Circulation | 38235591 | 2024 Apr 16 |
| Reversal of C9orf72 mutation-induced transcriptional dysregulation and pathology in cultured human neurons by allele-specific excision. | Sachdev, Aradhana; Gill, Kamaljot; Sckaff, Maria; Birk, Alisha M; Aladesuyi Arogundade, Olubankole; Brown, Katherine A; Chouhan, Runvir S; Issagholian-Lewin, Patrick Oliver; Patel, Esha; Watry, Hannah L; Bernardi, Mylinh T; Keough, Kathleen C; Tsai, Yu-Chih; Smith, Alec Simon Tulloch; Conklin, Bruce R; Clelland, Claire Dudley | Proceedings of the National Academy of Sciences of the United States of America | 38621131 | 2024 Apr 23 |
| Engineered IgM and IgG cleaving enzymes for mitigating antibody neutralization and complement activation in AAV gene transfer. | Smith, Timothy J; Elmore, Zachary C; Fusco, Robert M; Hull, Joshua A; Rosales, Alan; Martinez, Michele; Tarantal, Alice F; Asokan, Aravind | Molecular therapy : the journal of the American Society of Gene Therapy | 38715362 | 2024 Jul 03 |
| Modifying the Backbone Chemistry of PEG-Based Bottlebrush Block Copolymers for the Formation of Long-Circulating Nanoparticles. | Grundler, Julian; Whang, Chang-Hee; Shin, Kwangsoo; Savan, N Anna; Zhong, Mingjiang; Saltzman, W Mark | Advanced healthcare materials | 38734871 | 2024 Sep |
| Advances and challenges in modeling inherited peripheral neuropathies using iPSCs. | Van Lent, Jonas; Prior, Robert; Pérez Siles, Gonzalo; Cutrupi, Anthony N; Kennerson, Marina L; Vangansewinkel, Tim; Wolfs, Esther; Mukherjee-Clavin, Bipasha; Nevin, Zachary; Judge, Luke; Conklin, Bruce; Tyynismaa, Henna; Clark, Alex J; Bennett, David L; Van Den Bosch, Ludo; Saporta, Mario; Timmerman, Vincent | Experimental & molecular medicine | 38825644 | 2024 Jun |
| A Novel Humanized Mouse Model for HIV and Tuberculosis Co-infection Studies. | Bohórquez, José Alejandro; Adduri, Sitaramaraju; Ansari, Danish; John, Sahana; Florence, Jon; Adejare, Omoyeni; Singh, Gaurav; Konduru, Nagarjun; Jagannath, Chinnaswamy; Yi, Guohua | bioRxiv : the preprint server for biology | 38496484 | 2024 Mar 07 |
| Recommendations on fit-for-purpose criteria to establish quality management for microphysiological systems and for monitoring their reproducibility. | Pamies, David; Ekert, Jason; Zurich, Marie-Gabrielle; Frey, Olivier; Werner, Sophie; Piergiovanni, Monica; Freedman, Benjamin S; Keong Teo, Adrian Kee; Erfurth, Hendrik; Reyes, Darwin R; Loskill, Peter; Candarlioglu, Pelin; Suter-Dick, Laura; Wang, Shan; Hartung, Thomas; Coecke, Sandra; Stacey, Glyn N; Wagegg, Beren Atac; Dehne, Eva-Maria; Pistollato, Francesca; Leist, Marcel | Stem cell reports | 38959889 | 2024 Jul 02 |
| Systematic multi-trait AAV capsid engineering for efficient gene delivery. | Eid, Fatma-Elzahraa; Chen, Albert T; Chan, Ken Y; Huang, Qin; Zheng, Qingxia; Tobey, Isabelle G; Pacouret, Simon; Brauer, Pamela P; Keyes, Casey; Powell, Megan; Johnston, Jencilin; Zhao, Binhui; Lage, Kasper; Tarantal, Alice F; Chan, Yujia A; Deverman, Benjamin E | Nature communications | 39097583 | 2024 Aug 04 |
| Motion Blur Microscopy. | Goreke, Utku; Gonzales, Ayesha; Shipley, Brandon; Tincher, Madeleine; Sharma, Oshin; Wulftange, William; Man, Yuncheng; An, Ran; Hinczewski, Michael; Gurkan, Umut A | bioRxiv : the preprint server for biology | 37873474 | 2024 May 16 |
| Precision Enhancement of CAR-NK Cells through Non-Viral Engineering and Highly Multiplexed Base Editing. | Wang, Minjing; Krueger, Joshua B; Gilkey, Alexandria K; Stelljes, Erin M; Kluesner, Mitchell G; Pomeroy, Emily J; Skeate, Joseph G; Slipek, Nicholas J; Lahr, Walker S; Vázquez, Patricia N Claudio; Zhao, Yueting; Eaton, Ella J; Laoharawee, Kanut; Webber, Beau R; Moriarity, Branden S | bioRxiv : the preprint server for biology | 38496503 | 2024 Mar 08 |
| Selective haematological cancer eradication with preserved haematopoiesis. | Garaudé, Simon; Marone, Romina; Lepore, Rosalba; Devaux, Anna; Beerlage, Astrid; Seyres, Denis; Dell' Aglio, Alessandro; Juskevicius, Darius; Zuin, Jessica; Burgold, Thomas; Wang, Sisi; Katta, Varun; Manquen, Garret; Li, Yichao; Larrue, Clément; Camus, Anna; Durzynska, Izabela; Wellinger, Lisa C; Kirby, Ian; Van Berkel, Patrick H; Kunz, Christian; Tamburini, Jérôme; Bertoni, Francesco; Widmer, Corinne C; Tsai, Shengdar Q; Simonetta, Federico; Urlinger, Stefanie; Jeker, Lukas T | Nature | 38778101 | 2024 Jun |
| Multiscale engineering of brain organoids for disease modeling. | Xu, Cong; Alameri, Alia; Leong, Wei; Johnson, Emily; Chen, Zaozao; Xu, Bin; Leong, Kam W | Advanced drug delivery reviews | 38810702 | 2024 Jul |
| A prime editor mouse to model a broad spectrum of somatic mutations in vivo. | Ely, Zackery A; Mathey-Andrews, Nicolas; Naranjo, Santiago; Gould, Samuel I; Mercer, Kim L; Newby, Gregory A; Cabana, Christina M; Rideout 3rd, William M; Jaramillo, Grissel Cervantes; Khirallah, Jennifer M; Holland, Katie; Randolph, Peyton B; Freed-Pastor, William A; Davis, Jessie R; Kulstad, Zachary; Westcott, Peter M K; Lin, Lin; Anzalone, Andrew V; Horton, Brendan L; Pattada, Nimisha B; Shanahan, Sean-Luc; Ye, Zhongfeng; Spranger, Stefani; Xu, Qiaobing; Sánchez-Rivera, Francisco J; Liu, David R; Jacks, Tyler | Nature biotechnology | 37169967 | 2024 Mar |
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| Lipid nanoparticle-mediated codelivery of Cas9 mRNA and single-guide RNA achieves liver-specific in vivo genome editing of Angptl3. | Qiu, Min; Glass, Zachary; Chen, Jinjin; Haas, Mary; Jin, Xin; Zhao, Xuewei; Rui, Xuehui; Ye, Zhongfeng; Li, Yamin; Zhang, Feng; Xu, Qiaobing | Proceedings of the National Academy of Sciences of the United States of America | 33649229 | 2021 Mar 09 |
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| Three-dimensional tissue-engineered human skeletal muscle model of Pompe disease. | Wang, Jason; Zhou, Chris J; Khodabukus, Alastair; Tran, Sabrina; Han, Sang-Oh; Carlson, Aaron L; Madden, Lauran; Kishnani, Priya S; Koeberl, Dwight D; Bursac, Nenad | Communications biology | 33953320 | 2021 05 05 |
| 3D cell culture models: Drug pharmacokinetics, safety assessment, and regulatory consideration. | Wang, Hongbing; Brown, Paul C; Chow, Edwin C Y; Ewart, Lorna; Ferguson, Stephen S; Fitzpatrick, Suzanne; Freedman, Benjamin S; Guo, Grace L; Hedrich, William; Heyward, Scott; Hickman, James; Isoherranen, Nina; Li, Albert P; Liu, Qi; Mumenthaler, Shannon M; Polli, James; Proctor, William R; Ribeiro, Alexandre; Wang, Jian-Ying; Wange, Ronald L; Huang, Shiew-Mei | Clinical and translational science | 33982436 | 2021 09 |
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| Vectored Immunotherapeutics for Infectious Diseases: Can rAAVs Be The Game Changers for Fighting Transmissible Pathogens? | Zhan, Wei; Muhuri, Manish; Tai, Phillip W L; Gao, Guangping | Frontiers in immunology | 34046041 | 2021 |
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| Escaping the endosome: assessing cellular trafficking mechanisms of non-viral vehicles. | Xu, Emily; Saltzman, W Mark; Piotrowski-Daspit, Alexandra S | Journal of controlled release : official journal of the Controlled Release Society | 34077782 | 2021 07 10 |
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| Functional correction of CFTR mutations in human airway epithelial cells using adenine base editors. | Krishnamurthy, Sateesh; Traore, Soumba; Cooney, Ashley L; Brommel, Christian M; Kulhankova, Katarina; Sinn, Patrick L; Newby, Gregory A; Liu, David R; McCray, Paul B | Nucleic acids research | 34520545 | 2021 Oct 11 |
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| Organoid-on-a-chip model of human ARPKD reveals mechanosensing pathomechanisms for drug discovery. | Hiratsuka, Ken; Miyoshi, Tomoya; Kroll, Katharina T; Gupta, Navin R; Valerius, M Todd; Ferrante, Thomas; Yamashita, Michifumi; Lewis, Jennifer A; Morizane, Ryuji | Science advances | 36129975 | 2022 09 23 |
| Engineering multicellular living systems-A Keystone Symposia report. | Cable, Jennifer; Arlotta, Paola; Parker, Kevin Kit; Hughes, Alex J; Goodwin, Katharine; Mummery, Christine L; Kamm, Roger D; Engle, Sandra J; Tagle, Danilo A; Boj, Sylvia F; Stanton, Alice E; Morishita, Yoshihiro; Kemp, Melissa L; Norfleet, Dennis A; May, | Annals of the New York Academy of Sciences | 36177947 | 2022 Sep 30 |
| Direct ITR-to-ITR Nanopore Sequencing of AAV Vector Genomes. | Namkung, Suk; Tran, Ngoc Tam; Manokaran, Sangeetha; He, Ran; Su, Qin; Xie, Jun; Gao, Guangping; Tai, Phillip W L | Human gene therapy | 36178359 | 2022 Nov |
| In vivo correction of cystic fibrosis mediated by PNA nanoparticles. | Piotrowski-Daspit, Alexandra S; Barone, Christina; Lin, Chun-Yu; Deng, Yanxiang; Wu, Douglas; Binns, Thomas C; Xu, Emily; Ricciardi, Adele S; Putman, Rachael; Garrison, Alannah; Nguyen, Richard; Gupta, Anisha; Fan, Rong; Glazer, Peter M; Saltzman, W Mark; Egan, Marie E | Science advances | 36197984 | 2022 Oct 07 |
| Cross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome editing. | Gonzalez, Trevor J; Simon, Katherine E; Blondel, Leo O; Fanous, Marco M; Roger, Angela L; Maysonet, Maribel Santiago; Devlin, Garth W; Smith, Timothy J; Oh, Daniel K; Havlik, L Patrick; Castellanos Rivera, Ruth M; Piedrahita, Jorge A; ElMallah, Mai K; Ger | Nature communications | 36210364 | 2022 Oct 10 |
| EZ Clear for simple, rapid, and robust mouse whole organ clearing. | Hsu, Chih-Wei; Cerda, Juan; Kirk, Jason M; Turner, Williamson D; Rasmussen, Tara L; Flores Suarez, Carlos P; Dickinson, Mary E; Wythe, Joshua D | eLife | 36218247 | 2022 Oct 11 |
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| Translating musculoskeletal bioengineering into tissue regeneration therapies. | Khodabukus, Alastair; Guyer, Tyler; Moore, Axel C; Stevens, Molly M; Guldberg, Robert E; Bursac, Nenad | Science translational medicine | 36223445 | 2022 Oct 12 |
| Nucleic Acid Delivery to the Vascular Endothelium. | Reschke, Melanie; Piotrowski-Daspit, Alexandra S; Pober, Jordan S; Saltzman, W Mark | Molecular pharmaceutics | 36251765 | 2022 Dec 05 |
| Unadjuvanted intranasal spike vaccine elicits protective mucosal immunity against sarbecoviruses. | Mao, Tianyang; Israelow, Benjamin; Peña-Hernández, Mario A; Suberi, Alexandra; Zhou, Liqun; Luyten, Sophia; Reschke, Melanie; Dong, Huiping; Homer, Robert J; Saltzman, W Mark; Iwasaki, Akiko | Science (New York, N.Y.) | 36302057 | 2022 Nov 25 |
| Adult human kidney organoids originate from CD24+ cells and represent an advanced model for adult polycystic kidney disease. | Xu, Yaoxian; Kuppe, Christoph; Perales-Patón, Javier; Hayat, Sikander; Kranz, Jennifer; Abdallah, Ali T; Nagai, James; Li, Zhijian; Peisker, Fabian; Saritas, Turgay; Halder, Maurice; Menzel, Sylvia; Hoeft, Konrad; Kenter, Annegien; Kim, Hyojin; van Roeyen, Claudia R C; Lehrke, Michael; Moellmann, Julia; Speer, Thimoteus; Buhl, Eva M; Hoogenboezem, Remco; Boor, Peter; Jansen, Jitske; Knopp, Cordula; Kurth, Ingo; Smeets, Bart; Bindels, Eric; Reinders, Marlies E J; Baan, Carla; Gribnau, Joost; Hoorn, Ewout J; Steffens, Joachim; Huber, Tobias B; Costa, Ivan; Floege, Jürgen; Schneider, Rebekka K; Saez-Rodriguez, Julio; Freedman, Benjamin S; Kramann, Rafael | Nature genetics | 36303074 | 2022 Oct 27 |
| Prime editing for precise and highly versatile genome manipulation. | Chen, Peter J; Liu, David R | Nature reviews. Genetics | 36344749 | 2023 Mar |
| Evolution of an adenine base editor into a small, efficient cytosine base editor with low off-target activity. | Neugebauer, Monica E; Hsu, Alvin; Arbab, Mandana; Krasnow, Nicholas A; McElroy, Amber N; Pandey, Smriti; Doman, Jordan L; Huang, Tony P; Raguram, Aditya; Banskota, Samagya; Newby, Gregory A; Tolar, Jakub; Osborn, Mark J; Liu, David R | Nature biotechnology | 36357719 | 2022 Nov 10 |
| LPA disruption with AAV-CRISPR potently lowers plasma apo(a) in transgenic mouse model: A proof-of-concept study. | Doerfler, Alexandria M; Park, So Hyun; Assini, Julia M; Youssef, Amer; Saxena, Lavanya; Yaseen, Adam B; De Giorgi, Marco; Chuecos, Marcel; Hurley, Ayrea E; Li, Ang; Marcovina, Santica M; Bao, Gang; Boffa, Michael B; Koschinsky, Marlys L; Lagor, William R | Molecular therapy. Methods & clinical development | 36381302 | 2022 Dec 08 |
| Production and characterization of virus-free, CRISPR-CAR T cells capable of inducing solid tumor regression. | Mueller, Katherine P; Piscopo, Nicole J; Forsberg, Matthew H; Saraspe, Louise A; Das, Amritava; Russell, Brittany; Smerchansky, Madeline; Cappabianca, Dan; Shi, Lei; Shankar, Keerthana; Sarko, Lauren; Khajanchi, Namita; La Vonne Denne, Nina; Ramamurthy, A | Journal for immunotherapy of cancer | 36382633 | 2022 Sep |
| ABE8e adenine base editor precisely and efficiently corrects a recurrent COL7A1 nonsense mutation. | Sheriff, Adam; Guri, Ina; Zebrowska, Paulina; Llopis-Hernandez, Virginia; Brooks, Imogen R; Tekkela, Stavroula; Subramaniam, Kavita; Gebrezgabher, Ruta; Naso, Gaetano; Petrova, Anastasia; Balon, Katarzyna; Onoufriadis, Alexandros; Kujawa, Dorota; Kotulska, Martyna; Newby, Gregory; Łaczmański, Łukasz; Liu, David R; McGrath, John A; Jacków, Joanna | Scientific reports | 36385635 | 2022 Nov 16 |
| Compact zinc finger base editors that edit mitochondrial or nuclear DNA in vitro and in vivo. | Willis, Julian C W; Silva-Pinheiro, Pedro; Widdup, Lily; Minczuk, Michal; Liu, David R | Nature communications | 36418298 | 2022 Nov 23 |
| Diverse virus-encoded CRISPR-Cas systems include streamlined genome editors. | Al-Shayeb, Basem; Skopintsev, Petr; Soczek, Katarzyna M; Stahl, Elizabeth C; Li, Zheng; Groover, Evan; Smock, Dylan; Eggers, Amy R; Pausch, Patrick; Cress, Brady F; Huang, Carolyn J; Staskawicz, Brian; Savage, David F; Jacobsen, Steven E; Banfield, Jillia | Cell | 36423580 | 2022 Nov 23 |
| Substituting racemic ionizable lipids with stereopure ionizable lipids can increase mRNA delivery. | Da Silva Sanchez, Alejandro J; Zhao, Kun; Huayamares, Sebastian G; Hatit, Marine Z C; Lokugamage, Melissa P; Loughrey, David; Dobrowolski, Curtis; Wang, Shuaishuai; Kim, Hyejin; Paunovska, Kalina; Kuzminich, Yanina; Dahlman, James E | Journal of controlled release : official journal of the Controlled Release Society | 36423872 | 2022 Nov 30 |
| Drag-and-drop genome insertion of large sequences without double-strand DNA cleavage using CRISPR-directed integrases. | Yarnall, Matthew T N; Ioannidi, Eleonora I; Schmitt-Ulms, Cian; Krajeski, Rohan N; Lim, Justin; Villiger, Lukas; Zhou, Wenyuan; Jiang, Kaiyi; Garushyants, Sofya K; Roberts, Nathaniel; Zhang, Liyang; Vakulskas, Christopher A; Walker 2nd, John A; Kadina, Anastasia P; Zepeda, Adrianna E; Holden, Kevin; Ma, Hong; Xie, Jun; Gao, Guangping; Foquet, Lander; Bial, Greg; Donnelly, Sara K; Miyata, Yoshinari; Radiloff, Daniel R; Henderson, Jordana M; Ujita, Andrew; Abudayyeh, Omar O; Gootenberg, Jonathan S | Nature biotechnology | 36424489 | 2023 Apr |
| Cyclin G1 induces maladaptive proximal tubule cell dedifferentiation and renal fibrosis through CDK5 activation. | Taguchi, Kensei; Elias, Bertha C; Sugahara, Sho; Sant, Snehal; Freedman, Benjamin S; Waikar, Sushrut S; Pozzi, Ambra; Zent, Roy; Harris, Raymond C; Parikh, Samir M; Brooks, Craig R | The Journal of clinical investigation | 36453545 | 2022 Dec 01 |
| Reframing the perception of outliers and negative data in translational research. | Emborg, Marina E | Brain research bulletin | 36464129 | 2023 Jan |
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| Efficient in vivo neuronal genome editing in the mouse brain using nanocapsules containing CRISPR-Cas9 ribonucleoproteins. | Metzger, Jeanette M; Wang, Yuyuan; Neuman, Samuel S; Snow, Kathy J; Murray, Stephen A; Lutz, Cathleen M; Bondarenko, Viktoriya; Felton, Jesi; Gimse, Kirstan; Xie, Ruosen; Li, Dongdong; Zhao, Yi; Flowers, Matthew T; Simmons, Heather A; Roy, Subhojit; Saha, Krishanu; Levine, Jon E; Emborg, Marina E; Gong, Shaoqin | Biomaterials | 36527789 | 2022 Dec 13 |
| Emerging immunomodulatory strategies for cell therapeutics. | Chua, Corrine Ying Xuan; Jiang, Allen Yujie; Eufrásio-da-Silva, Tatiane; Dolatshahi-Pirouz, Alireza; Langer, Robert; Orive, Gorka; Grattoni, Alessandro | Trends in biotechnology | 36549959 | 2023 Mar |
| Glucose absorption drives cystogenesis in a human organoid-on-chip model of polycystic kidney disease. | Li, Sienna R; Gulieva, Ramila E; Helms, Louisa; Cruz, Nelly M; Vincent, Thomas; Fu, Hongxia; Himmelfarb, Jonathan; Freedman, Benjamin S | Nature communications | 36564419 | 2022 Dec 23 |
| Rigid respiration: fulminant pulmonary fibrosis after COVID-19. | Meyerholz, David K | EBioMedicine | 36580850 | 2023 Jan |
| In vivo editing of the pan-endothelium by immunity evading simian adenoviral vector. | Lorincz, Reka; Alvarez, Aluet Borrego; Walkey, Christopher J; Mendonça, Samir A; Lu, Zhi Hong; Martinez, Alexa E; Ljungberg, Cecilia; Heaney, Jason D; Lagor, William R; Curiel, David T | Biomedicine & pharmacotherapy = Biomedecine & pharmacotherapie | 36587560 | 2023 Feb |
| In Vivo Cellular Magnetic Imaging: Labeled vs. Unlabeled Cells. | Bulte, Jeff W M; Wang, Chao; Shakeri-Zadeh, Ali | Advanced functional materials | 36589903 | 2022 Dec 09 |
| Accounting for diversity in the design of CRISPR-based therapeutic genome editing. | Saha, Krishanu | Nature genetics | 36593304 | 2023 Jan |
| Synthetic Biology Design as a Paradigm Shift toward Manufacturing Affordable Adeno-Associated Virus Gene Therapies. | Collins, Logan Thrasher; Ponnazhagan, Selvarangan; Curiel, David T | ACS synthetic biology | 36627108 | 2023 Jan 20 |
| Phenylboronic Acid-Functionalized Polyplexes Tailored to Oral CRISPR Delivery. | Yoshinaga, Naoto; Zhou, Joyce K; Xu, Cong; Quek, Chai Hoon; Zhu, Yuefei; Tang, Ding; Hung, Lin Yung; Najjar, Sarah A; Shiu, Chin Ying Angela; Margolis, Kara Gross; Lao, Yeh-Hsing; Leong, Kam W | Nano letters | 36648291 | 2023 Feb 08 |
| Clinical progress in genome-editing technology and in vivo delivery techniques. | Khirallah, Jennifer; Eimbinder, Maximilan; Li, Yamin; Xu, Qiaobing | Trends in genetics : TIG | 36669950 | 2023 Jan 18 |
| CRISPR-Cas9-AAV versus lentivector transduction for genome modification of X-linked severe combined immunodeficiency hematopoietic stem cells. | Brault, Julie; Liu, Taylor; Liu, Siyuan; Lawson, Amanda; Choi, Uimook; Kozhushko, Nikita; Bzhilyanskaya, Vera; Pavel-Dinu, Mara; Meis, Ronald J; Eckhaus, Michael A; Burkett, Sandra S; Bosticardo, Marita; Kleinstiver, Benjamin P; Notarangelo, Luigi D; Lazzarotto, Cicera R; Tsai, Shengdar Q; Wu, Xiaolin; Dahl, Gary A; Porteus, Matthew H; Malech, Harry L; De Ravin, Suk See | Frontiers in immunology | 36685559 | 2022 |
| Precise transcript targeting by CRISPR-Csm complexes. | Colognori, David; Trinidad, Marena; Doudna, Jennifer A | Nature biotechnology | 36690762 | 2023 Sep |
| The Transcriptional Response to Lung-Targeting Lipid Nanoparticles in Vivo. | Radmand, Afsane; Lokugamage, Melissa P; Kim, Hyejin; Dobrowolski, Curtis; Zenhausern, Ryan; Loughrey, David; Huayamares, Sebastian G; Hatit, Marine Z C; Ni, Huanzhen; Del Cid, Ada; Da Silva Sanchez, Alejandro J; Paunovska, Kalina; Schrader Echeverri, Elisa; Shajii, Aram; Peck, Hannah; Santangelo, Philip J; Dahlman, James E | Nano letters | 36701517 | 2023 Feb 08 |
| Base editing as a genetic treatment for spinal muscular atrophy. | Alves, Christiano R R; Ha, Leillani L; Yaworski, Rebecca; Lazzarotto, Cicera R; Christie, Kathleen A; Reilly, Aoife; Beauvais, Ariane; Doll, Roman M; de la Cruz, Demitri; Maguire, Casey A; Swoboda, Kathryn J; Tsai, Shengdar Q; Kothary, Rashmi; Kleinstiver, Benjamin P | bioRxiv : the preprint server for biology | 36711797 | 2023 Jan 21 |
| Focused ultrasound-mediated brain genome editing. | Leong, Kam; Lao, Yeh-Hsing; Ji, Robin; Zhou, Joyce; Snow, Kathy; Kwon, Nancy; Saville, Ethan; He, Siyu; Chauhan, Shradha; Chi, Chun-Wei; Datta, Malika; Zhang, Hairong; Quek, Chai Hoon; Cai, Sarah; Li, Mingqiang; Gaitan, Yaned; Bechtel, Lawrence; Wu, Shih-Ying; Lutz, Cathleen; Tomer, Raju; Murray, Stephen; Chavez, Alejandro; Konofagou, Elisa | Research square | 36712096 | 2023 Jan 20 |
| Functional maturation of kidney organoid tubules: Piezo1-mediated Ca2+ signaling. | Carrisoza-Gaytán, Rolando; Kroll, Katharina T; Hiratsuka, Ken; Gupta, Navin R; Morizane, Ryuji; Lewis, Jennifer A; Satlin, Lisa M | American journal of physiology. Cell physiology | 36745528 | 2023 Feb 06 |
| Efficient in vivo genome editing prevents hypertrophic cardiomyopathy in mice. | Reichart, Daniel; Newby, Gregory A; Wakimoto, Hiroko; Lun, Mingyue; Gorham, Joshua M; Curran, Justin J; Raguram, Aditya; DeLaughter, Daniel M; Conner, David A; Marsiglia, Júlia D C; Kohli, Sajeev; Chmatal, Lukas; Page, David C; Zabaleta, Nerea; Vandenberghe, Luk; Liu, David R; Seidman, Jonathan G; Seidman, Christine | Nature medicine | 36797483 | 2023 Feb |
| In vivo HSC prime editing rescues sickle cell disease in a mouse model. | Li, Chang; Georgakopoulou, Aphrodite; Newby, Gregory A; Chen, Peter J; Everette, Kelcee A; Paschoudi, Kiriaki; Vlachaki, Efthymia; Gil, Sucheol; Anderson, Anna K; Koob, Theodore; Huang, Lishan; Wang, Hongjie; Kiem, Hans-Peter; Liu, David R; Yannaki, Evangelia; Lieber, André | Blood | 36800642 | 2023 Apr 27 |
| Shuttle Peptide Delivers Base Editor RNPs to Rhesus Monkey Airway Epithelial Cells In Vivo. | Kulhankova, Katarina; Traore, Soumba; Cheng, Xue; Benk-Fortin, Hadrien; Hallée, Stéphanie; Harvey, Mario; Roberge, Joannie; Couture, Frédéric; Gross, Thomas; Newby, Gregory; Liu, David; Tarantal, Alice; Guay, David; McCray, Paul | Research square | 36824928 | 2023 Feb 17 |
| In utero delivery of mRNA to the heart, diaphragm and muscle with lipid nanoparticles. | Gao, Kewa; Li, Jie; Song, Hengyue; Han, Hesong; Wang, Yongheng; Yin, Boyan; Farmer, Diana L; Murthy, Niren; Wang, Aijun | Bioactive materials | 36844366 | 2023 Jul |
| A parathyroid hormone/salt-inducible kinase signaling axis controls renal vitamin D activation and organismal calcium homeostasis. | Yoon, Sung-Hee; Meyer, Mark B; Arevalo Rivas, Carlos; Tekguc, Murat; Zhang, Chengcheng; Wang, Jialiang S; Castro Andrade, Christian D; Strauss, Katelyn E; Sato, Tadatoshi; Benkusky, Nancy; Lee, Seong Min; Berdeaux, Rebecca; Foretz, Marc; Sundberg, Thomas B; Xavier, Ramnik J; Adelmann, Charles H; Brooks, Daniel J; Anselmo, Anthony; Sadreyev, Ruslan I; Rosales, Ivy A; Fisher, David E; Gupta, Navin; Morizane, Ryuji; Greka, Anna; Pike, J Wesley; Mannstadt, Michael; Wein, Marc N | The Journal of clinical investigation | 36862513 | 2023 Mar 02 |
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| The history, use, and challenges of therapeutic somatic cell and germline gene editing. | Ryu, Junghyun; Adashi, Eli Y; Hennebold, Jon D | Fertility and sterility | 36878350 | 2023 Sep |
| ATP/ADP biosensor organoids for drug nephrotoxicity assessment. | Susa, Koichiro; Kobayashi, Kenichi; Galichon, Pierre; Matsumoto, Takuya; Tamura, Akitoshi; Hiratsuka, Ken; Gupta, Navin R; Yazdi, Iman K; Bonventre, Joseph V; Morizane, Ryuji | Frontiers in cell and developmental biology | 36936695 | 2023 |
| Human T cell generation is restored in CD3δ severe combined immunodeficiency through adenine base editing. | McAuley, Grace E; Yiu, Gloria; Chang, Patrick C; Newby, Gregory A; Campo-Fernandez, Beatriz; Fitz-Gibbon, Sorel T; Wu, Xiaomeng; Kang, Sung-Hae L; Garibay, Amber; Butler, Jeffrey; Christian, Valentina; Wong, Ryan L; Everette, Kelcee A; Azzun, Anthony; Gelfer, Hila; Seet, Christopher S; Narendran, Aru; Murguia-Favela, Luis; Romero, Zulema; Wright, Nicola; Liu, David R; Crooks, Gay M; Kohn, Donald B | Cell | 36944331 | 2023 Mar 30 |
| Mitochondrial Base Editing: Recent Advances towards Therapeutic Opportunities. | Kar, Bibekananda; Castillo, Santiago R; Sabharwal, Ankit; Clark, Karl J; Ekker, Stephen C | International journal of molecular sciences | 36982871 | 2023 Mar 18 |
| Self-delivering CRISPR RNAs for AAV Co-delivery and Genome Editing in vivo. | Zhang, Han; Kelly, Karen; Lee, Jonathan; Echeverria, Dimas; Cooper, David; Panwala, Rebecca; Chen, Zexiang; Gaston, Nicholas; Newby, Gregory A; Xie, Jun; Liu, David R; Gao, Guangping; Wolfe, Scot A; Khvorova, Anastasia; Watts, Jonathan K; Sontheimer, Erik J | bioRxiv : the preprint server for biology | 36993169 | 2023 Mar 23 |
| Mitigation of chromosome loss in clinical CRISPR-Cas9-engineered T cells. | Tsuchida, Connor A; Brandes, Nadav; Bueno, Raymund; Trinidad, Marena; Mazumder, Thomas; Yu, Bingfei; Hwang, Byungjin; Chang, Christopher; Liu, Jamin; Sun, Yang; Hopkins, Caitlin R; Parker, Kevin R; Qi, Yanyan; Satpathy, Ansuman T; Stadtmauer, Edward A; Cate, Jamie H D; Eyquem, Justin; Fraietta, Joseph A; June, Carl H; Chang, Howard Y; Ye, Chun Jimmie; Doudna, Jennifer A | bioRxiv : the preprint server for biology | 36993359 | 2023 Mar 22 |
| Base editing rescue of spinal muscular atrophy in cells and in mice. | Arbab, Mandana; Matuszek, Zaneta; Kray, Kaitlyn M; Du, Ailing; Newby, Gregory A; Blatnik, Anton J; Raguram, Aditya; Richter, Michelle F; Zhao, Kevin T; Levy, Jonathan M; Shen, Max W; Arnold, W David; Wang, Dan; Xie, Jun; Gao, Guangping; Burghes, Arthur H M; Liu, David R | Science (New York, N.Y.) | 36996170 | 2023 Apr 21 |
| Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing. | Li, Bowen; Manan, Rajith Singh; Liang, Shun-Qing; Gordon, Akiva; Jiang, Allen; Varley, Andrew; Gao, Guangping; Langer, Robert; Xue, Wen; Anderson, Daniel | Nature biotechnology | 36997680 | 2023 Oct |
| Rescue of GM3 synthase deficiency by spatially controlled, rAAV-mediated ST3GAL5 delivery. | Yang, Huiya; Brown Jr, Robert H; Wang, Dan; Strauss, Kevin A; Gao, Guangping | JCI insight | 37014712 | 2023 May 08 |
| Adeno-Associated virus 8 delivers an immunomodulatory peptide to mouse liver more efficiently than to rat liver. | Wang, Yuqing; Hurley, Ayrea; De Giorgi, Marco; Tanner, Mark R; Hu, Rong-Chi; Pennington, Michael W; Lagor, William R; Beeton, Christine | PloS one | 37040361 | 2023 |
| CaMKIIδ gene editing- A base hit for the heart. | Walkey, Christopher J; Lagor, William R | The journal of cardiovascular aging | 37065676 | 2023 |
| Reprogramming by drug-like molecules leads to regeneration of cochlear hair cell-like cells in adult mice. | Quan, Yi-Zhou; Wei, Wei; Ergin, Volkan; Rameshbabu, Arun Prabhu; Huang, Mingqian; Tian, Chunjie; Saladi, Srinivas Vinod; Indzhykulian, Artur A; Chen, Zheng-Yi | Proceedings of the National Academy of Sciences of the United States of America | 37068229 | 2023 Apr 25 |
| Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice. | Everette, Kelcee A; Newby, Gregory A; Levine, Rachel M; Mayberry, Kalin; Jang, Yoonjeong; Mayuranathan, Thiyagaraj; Nimmagadda, Nikitha; Dempsey, Erin; Li, Yichao; Bhoopalan, Senthil Velan; Liu, Xiong; Davis, Jessie R; Nelson, Andrew T; Chen, Peter J; Sousa, Alexander A; Cheng, Yong; Tisdale, John F; Weiss, Mitchell J; Yen, Jonathan S; Liu, David R | Nature biomedical engineering | 37069266 | 2023 May |
| Strategies for Improving Vascularization in Kidney Organoids: A Review of Current Trends. | Konoe, Ran; Morizane, Ryuji | Biology | 37106704 | 2023 Mar 26 |
| HIV-Differentiated Metabolite N-Acetyl-L-Alanine Dysregulates Human Natural Killer Cell Responses to Mycobacterium tuberculosis Infection. | Yang, Baojun; Mukherjee, Tanmoy; Radhakrishnan, Rajesh; Paidipally, Padmaja; Ansari, Danish; John, Sahana; Vankayalapati, Ramakrishna; Tripathi, Deepak; Yi, Guohua | International journal of molecular sciences | 37108430 | 2023 Apr 14 |
| Massively parallel base editing to map variant effects in human hematopoiesis. | Martin-Rufino, Jorge D; Castano, Nicole; Pang, Michael; Grody, Emanuelle I; Joubran, Samantha; Caulier, Alexis; Wahlster, Lara; Li, Tongqing; Qiu, Xiaojie; Riera-Escandell, Anna Maria; Newby, Gregory A; Al'Khafaji, Aziz; Chaudhary, Santosh; Black, Susan; Weng, Chen; Munson, Glen; Liu, David R; Wlodarski, Marcin W; Sims, Kacie; Oakley, Jamie H; Fasano, Ross M; Xavier, Ramnik J; Lander, Eric S; Klein, Daryl E; Sankaran, Vijay G | Cell | 37137305 | 2023 May 25 |
| Efficient prime editing in mouse brain, liver and heart with dual AAVs. | Davis, Jessie R; Banskota, Samagya; Levy, Jonathan M; Newby, Gregory A; Wang, Xiao; Anzalone, Andrew V; Nelson, Andrew T; Chen, Peter J; Hennes, Andrew D; An, Meirui; Roh, Heejin; Randolph, Peyton B; Musunuru, Kiran; Liu, David R | Nature biotechnology | 37142705 | 2023 May 04 |
| Peptide-mediated delivery of CRISPR enzymes for the efficient editing of primary human lymphocytes. | Foss, Dana V; Muldoon, Joseph J; Nguyen, David N; Carr, Daniel; Sahu, Srishti U; Hunsinger, John M; Wyman, Stacia K; Krishnappa, Netravathi; Mendonsa, Rima; Schanzer, Elaine V; Shy, Brian R; Vykunta, Vivasvan S; Allain, Vincent; Li, Zhongmei; Marson, Alexander; Eyquem, Justin; Wilson, Ross C | Nature biomedical engineering | 37147433 | 2023 May |
| Renewal of oligodendrocyte lineage reverses dysmyelination and CNS neurodegeneration through corrected N-acetylaspartate metabolism. | Lotun, Anoushka; Li, Danning; Xu, Hongxia; Su, Qin; Tuncer, Serafettin; Sanmiguel, Julio; Mooney, Morgan; Baer, Christina E; Ulbrich, Russell; Eyles, Stephen J; Strittmatter, Lara; Hayward, Lawrence J; Gessler, Dominic J; Gao, Guangping | Progress in neurobiology | 37149081 | 2023 Jul |
| Genome-wide profiling of prime editor off-target sites in vitro and in vivo using PE-tag. | Liang, Shun-Qing; Liu, Pengpeng; Ponnienselvan, Karthikeyan; Suresh, Sneha; Chen, Zexiang; Kramme, Christian; Chatterjee, Pranam; Zhu, Lihua Julie; Sontheimer, Erik J; Xue, Wen; Wolfe, Scot A | Nature methods | 37156841 | 2023 Jun |
| A Multifunctional and Highly Adaptable Reporter System for CRISPR/Cas Editing. | Wettengel, Jochen M; Hansen-Palmus, Lea; Yusova, Sofiya; Rust, Lauren; Biswas, Sreya; Carson, Julien; Ryu, Junghyun; Bimber, Benjamin N; Hennebold, Jon D; Burwitz, Benjamin J | International journal of molecular sciences | 37175977 | 2023 May 05 |
| Genetic background in the rat affects endocrine and metabolic outcomes of bisphenol F exposure. | Wagner, Valerie A; Holl, Katie L; Clark, Karen C; Reho, John J; Dwinell, Melinda R; Lehmler, Hans-Joachim; Raff, Hershel; Grobe, Justin L; Kwitek, Anne E | Toxicological sciences : an official journal of the Society of Toxicology | 37191987 | 2023 Jun 28 |
| Orthogonal CRISPR screens to identify transcriptional and epigenetic regulators of human CD8 T cell function. | McCutcheon, Sean R; Swartz, Adam M; Brown, Michael C; Barrera, Alejandro; Amador, Christian McRoberts; Siklenka, Keith; Humayun, Lucas; Isaacs, James M; Reddy, Timothy E; Nair, Smita; Antonia, Scott; Gersbach, Charles A | bioRxiv : the preprint server for biology | 37205457 | 2023 May 01 |
| Rescue of auditory function by a single administration of AAV-TMPRSS3 gene therapy in aged mice of human recessive deafness DFNB8. | Du, Wan; Ergin, Volkan; Loeb, Corena; Huang, Mingqian; Silver, Stewart; Armstrong, Ariel Miura; Huang, Zaohua; Gurumurthy, Channabasavaiah B; Staecker, Hinrich; Liu, Xuezhong; Chen, Zheng-Yi | Molecular therapy : the journal of the American Society of Gene Therapy | 37244253 | 2023 May 22 |
| Reducing the inherent auto-inhibitory interaction within the pegRNA enhances prime editing efficiency. | Ponnienselvan, Karthikeyan; Liu, Pengpeng; Nyalile, Thomas; Oikemus, Sarah; Maitland, Stacy A; Lawson, Nathan D; Luban, Jeremy; Wolfe, Scot A | Nucleic acids research | 37246708 | 2023 Jul 21 |
| Liver directed adeno-associated viral vectors to treat metabolic disease. | Chuecos, Marcel A; Lagor, William R | Journal of inherited metabolic disease | 37254440 | 2023 May 30 |
| Template-jumping prime editing enables large insertion and exon rewriting in vivo. | Zheng, Chunwei; Liu, Bin; Dong, Xiaolong; Gaston, Nicholas; Sontheimer, Erik J; Xue, Wen | Nature communications | 37291100 | 2023 Jun 08 |
| Rapid and definitive treatment of phenylketonuria in variant-humanized mice with corrective editing. | Brooks, Dominique L; Carrasco, Manuel J; Qu, Ping; Peranteau, William H; Ahrens-Nicklas, Rebecca C; Musunuru, Kiran; Alameh, Mohamad-Gabriel; Wang, Xiao | Nature communications | 37301931 | 2023 Jun 10 |
| Mercapto-pyrimidines are reversible covalent inhibitors of the papain-like protease (PLpro) and inhibit SARS-CoV-2 (SCoV-2) replication. | Bajaj, Teena; Wehri, Eddie; Suryawanshi, Rahul K; King, Elizabeth; Pardeshi, Kundan Singh; Behrouzi, Kamyar; Khodabakhshi, Zahra; Schulze-Gahmen, Ursula; Kumar, G Renuka; Mofrad, Mohammad R K; Nomura, Daniel K; Ott, Melanie; Schaletzky, Julia; Murthy, Niren | RSC advances | 37312993 | 2023 Jun 09 |
| Scalable projected Light Sheet Microscopy for high-resolution imaging of large samples. | Chen, Yannan; Gong, Cheng; Chauhan, Shradha; De La Cruz, Estanislao Daniel; Datta, Malika S; Tomer, Raju | bioRxiv : the preprint server for biology | 37333196 | 2023 Jun 05 |
| Querying Recombination Junctions of Replication-Competent Adeno-Associated Viruses in Gene Therapy Vector Preparations with Single Molecule, Real-Time Sequencing. | Yip, Mitchell; Chen, Jing; Zhi, Yan; Tran, Ngoc Tam; Namkung, Suk; Pastor, Eric; Gao, Guangping; Tai, Phillip W L | Viruses | 37376529 | 2023 May 24 |
| Utilizing directed evolution to interrogate and optimize CRISPR/Cas guide RNA scaffolds. | Bush, Korie; Corsi, Giulia I; Yan, Amy C; Haynes, Keith; Layzer, Juliana M; Zhou, Jonathan H; Llanga, Telmo; Gorodkin, Jan; Sullenger, Bruce A | Cell chemical biology | 37390831 | 2023 Aug 17 |
| Potent and uniform fetal hemoglobin induction via base editing. | Mayuranathan, Thiyagaraj; Newby, Gregory A; Feng, Ruopeng; Yao, Yu; Mayberry, Kalin D; Lazzarotto, Cicera R; Li, Yichao; Levine, Rachel M; Nimmagadda, Nikitha; Dempsey, Erin; Kang, Guolian; Porter, Shaina N; Doerfler, Phillip A; Zhang, Jingjing; Jang, Yoonjeong; Chen, Jingjing; Bell, Henry W; Crossley, Merlin; Bhoopalan, Senthil Velan; Sharma, Akshay; Tisdale, John F; Pruett-Miller, Shondra M; Cheng, Yong; Tsai, Shengdar Q; Liu, David R; Weiss, Mitchell J; Yen, Jonathan S | Nature genetics | 37400614 | 2023 Jul |
| Rational nanoparticle design: Optimization using insights from experiments and mathematical models. | Richfield, Owen; Piotrowski-Daspit, Alexandra S; Shin, Kwangsoo; Saltzman, W Mark | Journal of controlled release : official journal of the Controlled Release Society | 37442201 | 2023 Aug |
| Adenoviral vectors infect B lymphocytes in vivo. | Rice-Boucher, Paul J; Mendonça, Samir Andrade; Alvarez, Aluet Borrego; Sturtz, Alexandria J; Lorincz, Reka; Dmitriev, Igor P; Kashentseva, Elena A; Lu, Zhi Hong; Romano, Rosa; Selby, Mark; Pingale, Kunal; Curiel, David T | Molecular therapy : the journal of the American Society of Gene Therapy | 37452494 | 2023 Sep 06 |
| Targeting AAV vectors to the central nervous system by engineering capsid-receptor interactions that enable crossing of the blood-brain barrier. | Huang, Qin; Chen, Albert T; Chan, Ken Y; Sorensen, Hikari; Barry, Andrew J; Azari, Bahar; Zheng, Qingxia; Beddow, Thomas; Zhao, Binhui; Tobey, Isabelle G; Moncada-Reid, Cynthia; Eid, Fatma-Elzahraa; Walkey, Christopher J; Ljungberg, M Cecilia; Lagor, William R; Heaney, Jason D; Chan, Yujia A; Deverman, Benjamin E | PLoS biology | 37467291 | 2023 Jul |
| Gene editing therapeutics based on mRNA delivery. | Popovitz, Juliana; Sharma, Rohit; Hoshyar, Reyhane; Soo Kim, Beob; Murthy, Niren; Lee, Kunwoo | Advanced drug delivery reviews | 37516409 | 2023 Sep |
| Individually tailored spatial-spectral pulsed CEST MRI for ratiometric mapping of myocardial energetic species at 3T. | Ayala, Cindy; Luo, Huiwen; Godines, Kevin; Alghuraibawi, Wissam; Ahn, Sinyeob; Rehwald, Wolfgang; Grissom, William A; Vandsburger, Moriel H | Magnetic resonance in medicine | 37526176 | 2023 Dec |
| In vivo expansion of gene-targeted hepatocytes through transient inhibition of an essential gene. | De Giorgi, Marco; Park, So Hyun; Castoreno, Adam; Cao, Mingming; Hurley, Ayrea; Saxena, Lavanya; Chuecos, Marcel A; Walkey, Christopher J; Doerfler, Alexandria M; Furgurson, Mia N; Ljungberg, M Cecilia; Patel, Kalyani R; Hyde, Sarah; Chickering, Tyler; Lefebvre, Stephanie; Wassarman, Kelly; Miller, Patrick; Qin, June; Schlegel, Mark K; Zlatev, Ivan; Li, Rich Gang; Kim, Jong; Martin, James F; Bissig, Karl-Dimiter; Jadhav, Vasant; Bao, Gang; Lagor, William R | bioRxiv : the preprint server for biology | 37546995 | 2023 Jul 29 |
| Protospacer modification improves base editing of a canonical splice site variant and recovery of CFTR function in human airway epithelial cells. | Joynt, Anya T; Kavanagh, Erin W; Newby, Gregory A; Mitchell, Shakela; Eastman, Alice C; Paul, Kathleen C; Bowling, Alyssa D; Osorio, Derek L; Merlo, Christian A; Patel, Shivani U; Raraigh, Karen S; Liu, David R; Sharma, Neeraj; Cutting, Garry R | Molecular therapy. Nucleic acids | 37547293 | 2023 Sep 12 |
| Nonviral base editing of KCNJ13 mutation preserves vision in a model of inherited retinal channelopathy. | Kabra, Meha; Shahi, Pawan K; Wang, Yuyuan; Sinha, Divya; Spillane, Allison; Newby, Gregory A; Saxena, Shivani; Tong, Yao; Chang, Yu; Abdeen, Amr A; Edwards, Kimberly L; Theisen, Cole O; Liu, David R; Gamm, David M; Gong, Shaoqin; Saha, Krishanu; Pattnaik, Bikash R | The Journal of clinical investigation | 37561581 | 2023 Oct 02 |
| Focused ultrasound-mediated brain genome editing. | Lao, Yeh-Hsing; Ji, Robin; Zhou, Joyce K; Snow, Kathy J; Kwon, Nancy; Saville, Ethan; He, Siyu; Chauhan, Shradha; Chi, Chun-Wei; Datta, Malika S; Zhang, Hairong; Quek, Chai Hoon; Cai, S Sarah; Li, Mingqiang; Gaitan, Yaned; Bechtel, Lawrence; Wu, Shih-Ying; Lutz, Cathleen M; Tomer, Raju; Murray, Stephen A; Chavez, Alejandro; Konofagou, Elisa E; Leong, Kam W | Proceedings of the National Academy of Sciences of the United States of America | 37579143 | 2023 Aug 22 |
| CFTR-rich ionocytes mediate chloride absorption across airway epithelia. | Lei, Lei; Traore, Soumba; Romano Ibarra, Guillermo S; Karp, Philip H; Rehman, Tayyab; Meyerholz, David K; Zabner, Joseph; Stoltz, David A; Sinn, Patrick L; Welsh, Michael J; McCray Jr, Paul B; Thornell, Ian M | The Journal of clinical investigation | 37581935 | 2023 Oct 16 |
| Nonviral In Vivo Delivery of CRISPR-Cas9 Using Protein-Agnostic, High-Loading Porous Silicon and Polymer Nanoparticles. | Fletcher, R Brock; Stokes, Larry D; Kelly 3rd, Isom B; Henderson, Katelyn M; Vallecillo-Viejo, Isabel C; Colazo, Juan M; Wong, Benjamin V; Yu, Fang; d'Arcy, Richard; Struthers, Morgan N; Evans, Brian C; Ayers, Jacob; Castanon, Matthew; Weirich, Michael J; Reilly, Sarah K; Patel, Shrusti S; Ivanova, Yoanna I; Silvera Batista, Carlos A; Weiss, Sharon M; Gersbach, Charles A; Brunger, Jonathan M; Duvall, Craig L | ACS nano | 37582231 | 2023 Sep 12 |
| Treatment of monogenic and digenic dominant genetic hearing loss by CRISPR-Cas9 ribonucleoprotein delivery in vivo. | Tao, Yong; Lamas, Veronica; Du, Wan; Zhu, Wenliang; Li, Yiran; Whittaker, Madelynn N; Zuris, John A; Thompson, David B; Rameshbabu, Arun Prabhu; Shu, Yilai; Gao, Xue; Hu, Johnny H; Pei, Charles; Kong, Wei-Jia; Liu, Xuezhong; Wu, Hao; Kleinstiver, Benjamin P; Liu, David R; Chen, Zheng-Yi | Nature communications | 37582836 | 2023 Aug 15 |
| Polymer nanoparticles deliver mRNA to the lung for mucosal vaccination. | Suberi, Alexandra; Grun, Molly K; Mao, Tianyang; Israelow, Benjamin; Reschke, Melanie; Grundler, Julian; Akhtar, Laiba; Lee, Teresa; Shin, Kwangsoo; Piotrowski-Daspit, Alexandra S; Homer, Robert J; Iwasaki, Akiko; Suh, Hee-Won; Saltzman, W Mark | Science translational medicine | 37585505 | 2023 Aug 16 |
| Phage-assisted evolution and protein engineering yield compact, efficient prime editors. | Doman, Jordan L; Pandey, Smriti; Neugebauer, Monica E; An, Meirui; Davis, Jessie R; Randolph, Peyton B; McElroy, Amber; Gao, Xin D; Raguram, Aditya; Richter, Michelle F; Everette, Kelcee A; Banskota, Samagya; Tian, Kathryn; Tao, Y Allen; Tolar, Jakub; Osborn, Mark J; Liu, David R | Cell | 37657419 | 2023 Aug 31 |
| Trichostatin A for Efficient CRISPR-Cas9 Gene Editing of Human Pluripotent Stem Cells. | Molugu, Kaivalya; Khajanchi, Namita; Lazzarotto, Cicera R; Tsai, Shengdar Q; Saha, Krishanu | The CRISPR journal | 37676985 | 2023 Oct |
| Lipid Nanoparticles Deliver mRNA to the Brain after an Intracerebral Injection. | Tuma, Jan; Chen, Yu-Ju; Collins, Michael G; Paul, Abhik; Li, Jie; Han, Hesong; Sharma, Rohit; Murthy, Niren; Lee, Hye Young | Biochemistry | 37729550 | 2023 Dec 19 |
| Structure-guided AAV capsid evolution strategies for enhanced CNS gene delivery. | Gonzalez, Trevor J; Mitchell-Dick, Aaron; Blondel, Leo O; Fanous, Marco M; Hull, Joshua A; Oh, Daniel K; Moller-Tank, Sven; Castellanos Rivera, Ruth M; Piedrahita, Jorge A; Asokan, Aravind | Nature protocols | 37735235 | 2023 Nov |
| A brown fat-enriched adipokine, ASRA, is a leptin receptor antagonist that stimulates appetite. | Huang, Lei; Liu, Pengpeng; Du, Yong; Pan, Dongning; Lee, Alexandra; Wolfe, Scot A; Wang, Yong-Xu | bioRxiv : the preprint server for biology | 37745491 | 2023 Sep 12 |
| Navigating the kidney organoid: insights into assessment and enhancement of nephron function. | Tabibzadeh, Nahid; Satlin, Lisa M; Jain, Sanjay; Morizane, Ryuji | American journal of physiology. Renal physiology | 37767571 | 2023 Dec 01 |
| Assigning functionality to cysteines by base editing of cancer dependency genes. | Li, Haoxin; Ma, Tiantai; Remsberg, Jarrett R; Won, Sang Joon; DeMeester, Kristen E; Njomen, Evert; Ogasawara, Daisuke; Zhao, Kevin T; Huang, Tony P; Lu, Bingwen; Simon, Gabriel M; Melillo, Bruno; Schreiber, Stuart L; Lykke-Andersen, Jens; Liu, David R; Cravatt, Benjamin F | Nature chemical biology | 37783940 | 2023 Nov |
| Mitigation of chromosome loss in clinical CRISPR-Cas9-engineered T cells. | Tsuchida, Connor A; Brandes, Nadav; Bueno, Raymund; Trinidad, Marena; Mazumder, Thomas; Yu, Bingfei; Hwang, Byungjin; Chang, Christopher; Liu, Jamin; Sun, Yang; Hopkins, Caitlin R; Parker, Kevin R; Qi, Yanyan; Hofman, Laura; Satpathy, Ansuman T; Stadtmauer, Edward A; Cate, Jamie H D; Eyquem, Justin; Fraietta, Joseph A; June, Carl H; Chang, Howard Y; Ye, Chun Jimmie; Doudna, Jennifer A | Cell | 37794590 | 2023 Oct 12 |
| Redirecting AAV vectors to extrahepatic tissues. | Asokan, Aravind; Shen, Shen | Molecular therapy : the journal of the American Society of Gene Therapy | 37805712 | 2023 Dec 06 |
| A marmoset brain cell census reveals regional specialization of cellular identities. | Krienen, Fenna M; Levandowski, Kirsten M; Zaniewski, Heather; Del Rosario, Ricardo C H; Schroeder, Margaret E; Goldman, Melissa; Wienisch, Martin; Lutservitz, Alyssa; Beja-Glasser, Victoria F; Chen, Cindy; Zhang, Qiangge; Chan, Ken Y; Li, Katelyn X; Sharma, Jitendra; McCormack, Dana; Shin, Tay Won; Harrahill, Andrew; Nyase, Eric; Mudhar, Gagandeep; Mauermann, Abigail; Wysoker, Alec; Nemesh, James; Kashin, Seva; Vergara, Josselyn; Chelini, Gabriele; Dimidschstein, Jordane; Berretta, Sabina; Deverman, Benjamin E; Boyden, Ed; McCarroll, Steven A; Feng, Guoping | Science advances | 37824615 | 2023 Oct 13 |
| Avian influenza A viruses exhibit plasticity in sialylglycoconjugate receptor usage in human lung cells. | Liang, Chieh-Yu; Huang, Iris; Han, Julianna; Sownthirarajan, Boopathi; Kulhankova, Katarina; Murray, Nathan B; Taherzadeh, Mehrnoush; Archer-Hartmann, Stephanie; Pepi, Lauren; Manivasagam, Senthamizharasi; Plung, Jesse; Sturtz, Miranda; Yu, Yolanda; Vogel, Olivia A; Kandasamy, Matheswaran; Gourronc, Francoise A; Klingelhutz, Aloysius J; Choudhury, Biswa; Rong, Lijun; Perez, Jasmine T; Azadi, Parastoo; McCray Jr, Paul B; Neelamegham, Sriram; Manicassamy, Balaji | Journal of virology | 37843369 | 2023 Nov 30 |
| PCSK9 activation promotes early atherosclerosis in a vascular microphysiological system. | Lee, Jounghyun H; Shores, Kevin L; Breithaupt, Jason J; Lee, Caleb S; Fodera, Daniella M; Kwon, Jennifer B; Ettyreddy, Adarsh R; Myers, Kristin M; Evison, Benny J; Suchowerska, Alexandra K; Gersbach, Charles A; Leong, Kam W; Truskey, George A | APL bioengineering | 37854060 | 2023 Dec |
| The Power of the Heterogeneous Stock Rat Founder Strains in Modeling Metabolic Disease. | Wagner, Valerie A; Holl, Katie L; Clark, Karen C; Reho, John J; Lehmler, Hans-Joachim; Wang, Kai; Grobe, Justin L; Dwinell, Melinda R; Raff, Hershel; Kwitek, Anne E | Endocrinology | 37882530 | 2023 Nov 02 |
| Non-canonical amino acid incorporation into AAV5 capsid enhances lung transduction in mice. | Chang, Hao; Du, Ailing; Jiang, Jun; Ren, Lingzhi; Liu, Nan; Zhou, Xuntao; Liang, Jialing; Gao, Guangping; Wang, Dan | Molecular therapy. Methods & clinical development | 37886602 | 2023 Dec 14 |
| DNA recognition and induced genome modification by a hydroxymethyl-γ tail-clamp peptide nucleic acid. | Oyaghire, Stanley N; Quijano, Elias; Perera, J Dinithi R; Mandl, Hanna K; Saltzman, W Mark; Bahal, Raman; Glazer, Peter M | Cell reports. Physical science | 37920723 | 2023 Oct 18 |
| Efficient in vivo prime editing corrects the most frequent phenylketonuria variant, associated with high unmet medical need. | Brooks, Dominique L; Whittaker, Madelynn N; Qu, Ping; Musunuru, Kiran; Ahrens-Nicklas, Rebecca C; Wang, Xiao | American journal of human genetics | 37924808 | 2023 Dec 07 |
| Building blocks for better biorepositories in Africa. | Croxton, Talishiea; Jonathan, Emmanuel; Suleiman, Kareemah; Balogun, Olasinbo; Ozumba, Petronilla J; Aloyo, Sharley M; Nsubuga, Gideon; Kamulegeya, Rogers E; Newton, Lwanga; Mukisa, John; Kader, Mukthar; Damaneite, Vuyo; Nadoma, Sunji; Onyemata, Ezenwa James; Anzaku, Abbas Abel; Nasinghe, Emmanuel; Troyer, Jennifer; Joubert, Bonnie R; Beiswanger, Christine; Joloba, Moses L; Mayne, Elizabeth; Abimiku, Alash'le; H3Africa Biorepository PI Working Group | Genome medicine | 37932809 | 2023 Nov 06 |
| Human red blood cells release microvesicles with distinct sizes and protein composition that alter neutrophil phagocytosis. | de Oliveira Junior, Getulio Pereira; Welsh, Joshua A; Pinckney, Brandy; Palu, Cintia C; Lu, Shulin; Zimmerman, Alan; Barbosa, Raquel Hora; Sahu, Parul; Noshin, Maeesha; Gummuluru, Suryaram; Tigges, John; Jones, Jennifer Clare; Ivanov, Alexander R; Ghiran, Ionita C | Journal of extracellular biology | 37942280 | 2023 Nov |
| Transcriptional and epigenetic regulators of human CD8+ T cell function identified through orthogonal CRISPR screens. | McCutcheon, Sean R; Swartz, Adam M; Brown, Michael C; Barrera, Alejandro; McRoberts Amador, Christian; Siklenka, Keith; Humayun, Lucas; Ter Weele, Maria A; Isaacs, James M; Reddy, Timothy E; Allen, Andrew S; Nair, Smita K; Antonia, Scott J; Gersbach, Charles A | Nature genetics | 37945901 | 2023 Dec |
| Eukaryotic RNA-guided endonucleases evolved from a unique clade of bacterial enzymes. | Yoon, Peter H; Skopintsev, Petr; Shi, Honglue; Chen, LinXing; Adler, Benjamin A; Al-Shimary, Muntathar; Craig, Rory J; Loi, Kenneth J; DeTurk, Evan C; Li, Zheng; Amerasekera, Jasmine; Trinidad, Marena; Nisonoff, Hunter; Chen, Kai; Lahiri, Arushi; Boger, Ron; Jacobsen, Steve; Banfield, Jillian F; Doudna, Jennifer A | Nucleic acids research | 37971304 | 2023 Dec 11 |
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| Genomic characterization of SARS-CoV-2 from Uganda using MinION nanopore sequencing. | Kia, Praiscillia; Katagirya, Eric; Kakembo, Fredrick Elishama; Adera, Doreen Ato; Nsubuga, Moses Luutu; Yiga, Fahim; Aloyo, Sharley Melissa; Aujat, Brendah Ronah; Anguyo, Denis Foe; Katabazi, Fred Ashaba; Kigozi, Edgar; Joloba, Moses L; Kateete, David Patrick | Scientific reports | 37993530 | 2023 Nov 22 |
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