Program Publications
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 |
|---|---|---|---|---|
| Accounting for diversity in the design of CRISPR-based therapeutic genome editing. | Saha, Krishanu | Nature genetics | 36593304 | 2023 Jan |
| 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 | 2023 May |
| 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 |
| 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 |
| Rigid respiration: fulminant pulmonary fibrosis after COVID-19. | Meyerholz, David K | EBioMedicine | 36580850 | 2023 Jan |
| 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 |
| 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 |
| 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 | 2023 Jan |
| 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 |
| Multiomics characterization of mouse hepatoblastoma identifies yes-associated protein 1 target genes. | Rodríguez, Tomás C; Kwan, Suet-Yan; Smith, Jordan L; Dadafarin, Sina; Wu, Chern-Horng; Sontheimer, Erik J; Xue, Wen | Hepatology (Baltimore, Md.) | 35932276 | 2023 Jul 01 |
| 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 |
| Reframing the perception of outliers and negative data in translational research. | Emborg, Marina E | Brain research bulletin | 36464129 | 2023 Jan |
| 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 |
| Nanoparticle stereochemistry-dependent endocytic processing improves in vivo mRNA delivery. | Hatit, Marine Z C; Dobrowolski, Curtis N; Lokugamage, Melissa P; Loughrey, David; Ni, Huanzhen; Zurla, Chiara; Da Silva Sanchez, Alejandro J; Radmand, Afsane; Huayamares, Sebastian G; Zenhausern, Ryan; Paunovska, Kalina; Peck, Hannah E; Kim, Jinwhan; Sato, Manaka; Feldman, Jacob I; Rivera, Michael-Alexander; Cristian, Ana; Kim, YongTae; Santangelo, Philip J; Dahlman, James E | Nature chemistry | 36864143 | 2023 Apr |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| CaMKIIδ gene editing- A base hit for the heart. | Walkey, Christopher J; Lagor, William R | The journal of cardiovascular aging | 37065676 | 2023 |
| Functional maturation of kidney organoid tubules: PIEZO1-mediated Ca2+ signaling. | Carrisoza-Gaytan, 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 Mar 01 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 | 2023 Feb |
| 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 |
| 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 |
| Prime editing for precise and highly versatile genome manipulation. | Chen, Peter J; Liu, David R | Nature reviews. Genetics | 36344749 | 2023 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 | 2023 May 04 |
| 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 |
| 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 |
| 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 Mar |
| 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 |
| A parathyroid hormone/salt-inducible kinase signaling axis controls renal vitamin D activation and organismal calcium homeostasis. | Yoon, Sung-Hee; Meyer, Mark B; Arevalo, Carlos; Tekguc, Murat; Zhang, Chengcheng; Wang, Jialiang S; Castro Andrade, Christian D; Strauss, Katelyn; Sato, Tadatoshi; Benkusky, Nancy A; 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 May 01 |
| High-throughput continuous evolution of compact Cas9 variants targeting single-nucleotide-pyrimidine PAMs. | Huang, Tony P; Heins, Zachary J; Miller, Shannon M; Wong, Brandon G; Balivada, Pallavi A; Wang, Tina; Khalil, Ahmad S; Liu, David R | Nature biotechnology | 36076084 | 2023 Jan |
| 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 |
| In situ autofluorescence lifetime assay of a photoreceptor stimulus response in mouse retina and human retinal organoids. | Samimi, Kayvan; Pattnaik, Bikash R; Capowski, Elizabeth E; Saha, Krishanu; Gamm, David M; Skala, Melissa C | Biomedical optics express | 35781966 | 2022 Jun 01 |
| Myoblast deactivation within engineered human skeletal muscle creates a transcriptionally heterogeneous population of quiescent satellite-like cells. | Wang, Jason; Broer, Torie; Chavez, Taylor; Zhou, Chris J; Tran, Sabrina; Xiang, Yu; Khodabukus, Alastair; Diao, Yarui; Bursac, Nenad | Biomaterials | 35421801 | 2022 May |
| In vivo base editing rescues cone photoreceptors in a mouse model of early-onset inherited retinal degeneration. | Choi, Elliot H; Suh, Susie; Foik, Andrzej T; Leinonen, Henri; Newby, Gregory A; Gao, Xin D; Banskota, Samagya; Hoang, Thanh; Du, Samuel W; Dong, Zhiqian; Raguram, Aditya; Kohli, Sajeev; Blackshaw, Seth; Lyon, David C; Liu, David R; Palczewski, Krzysztof | Nature communications | 35383196 | 2022 Apr 05 |
| Prioritization of autoimmune disease-associated genetic variants that perturb regulatory element activity in T cells. | Mouri, Kousuke; Guo, Michael H; de Boer, Carl G; Lissner, Michelle M; Harten, Ingrid A; Newby, Gregory A; DeBerg, Hannah A; Platt, Winona F; Gentili, Matteo; Liu, David R; Campbell, Daniel J; Hacohen, Nir; Tewhey, Ryan; Ray, John P | Nature genetics | 35513721 | 2022 May |
| Therapeutic in vivo delivery of gene editing agents. | Raguram, Aditya; Banskota, Samagya; Liu, David R | Cell | 35798006 | 2022 Jul 21 |
| In vivo engineered B cells secrete high titers of broadly neutralizing anti-HIV antibodies in mice. | Nahmad, Alessio D; Lazzarotto, Cicera R; Zelikson, Natalie; Kustin, Talia; Tenuta, Mary; Huang, Deli; Reuveni, Inbal; Nataf, Daniel; Raviv, Yuval; Horovitz-Fried, Miriam; Dotan, Iris; Carmi, Yaron; Rosin-Arbesfeld, Rina; Nemazee, David; Voss, James E; Stern, Adi; Tsai, Shengdar Q; Barzel, Adi | Nature biotechnology | 35681059 | 2022 Aug |
| Modeling injury and repair in kidney organoids reveals that homologous recombination governs tubular intrinsic repair. | Gupta, Navin; Matsumoto, Takuya; Hiratsuka, Ken; Garcia Saiz, Edgar; Galichon, Pierre; Miyoshi, Tomoya; Susa, Koichiro; Tatsumoto, Narihito; Yamashita, Michifumi; Morizane, Ryuji | Science translational medicine | 35235339 | 2022 Mar 02 |
| Derivation of extra-embryonic and intra-embryonic macrophage lineages from human pluripotent stem cells. | Bredemeyer, Andrea L; Amrute, Junedh M; Koenig, Andrew L; Idol, Rachel A; He, Li; Luff, Stephanie A; Dege, Carissa; Leid, Jamison M; Schilling, Joel D; Hinson, J Travis; Dinauer, Mary C; Sturgeon, Christopher M; Lavine, Kory J | Development (Cambridge, England) | 35178561 | 2022 Apr 15 |
| Kidney organoids: a pioneering model for kidney diseases. | Tekguc, Murat; Gaal, Ronald C VAN; Uzel, Sebastien G M; Gupta, Navin; Riella, Leonardo V; Lewis, Jennifer A; Morizane, Ryuji | Translational research : the journal of laboratory and clinical medicine | 35750295 | 2022 Dec |
| CRISPR Library Screening in Cultured Cardiomyocytes. | DeLuca, Sophia; Bursac, Nenad | Methods in molecular biology (Clifton, N.J.) | 35618895 | 2022 |
| Differential microRNA profiles of intramuscular and secreted extracellular vesicles in human tissue-engineered muscle. | Vann, Christopher G; Zhang, Xin; Khodabukus, Alastair; Orenduff, Melissa C; Chen, Yu-Hsiu; Corcoran, David L; Truskey, George A; Bursac, Nenad; Kraus, Virginia B | Frontiers in physiology | 36091396 | 2022 |
| Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins. | Banskota, Samagya; Raguram, Aditya; Suh, Susie; Du, Samuel W; Davis, Jessie R; Choi, Elliot H; Wang, Xiao; Nielsen, Sarah C; Newby, Gregory A; Randolph, Peyton B; Osborn, Mark J; Musunuru, Kiran; Palczewski, Krzysztof; Liu, David R | Cell | 35021064 | 2022 Jan 20 |
| rAAV-based and intraprostatically delivered miR-34a therapeutics for efficient inhibition of prostate cancer progression. | Ai, Jianzhong; Li, Jia; Su, Qin; Ma, Hong; He, Ran; Wei, Qiang; Li, Hong; Gao, Guangping | Gene therapy | 34226687 | 2022 Aug |
| AAV5 delivery of CRISPR-Cas9 supports effective genome editing in mouse lung airway. | Liang, Shun-Qing; Walkey, Christopher J; Martinez, Alexa E; Su, Qin; Dickinson, Mary E; Wang, Dan; Lagor, William R; Heaney, Jason D; Gao, Guangping; Xue, Wen | Molecular therapy : the journal of the American Society of Gene Therapy | 34695545 | 2022 Jan 05 |
| Intestinal Deletion of 3-Hydroxy-3-Methylglutaryl-Coenzyme A Reductase Promotes Expansion of the Resident Stem Cell Compartment. | Doerfler, Alexandria M; Han, Jun; Jarrett, Kelsey E; Tang, Li; Jain, Antrix; Saltzman, Alexander; De Giorgi, Marco; Chuecos, Marcel; Hurley, Ayrea E; Li, Ang; Morand, Pauline; Ayala, Claudia; Goodlett, David R; Malovannaya, Anna; Martin, James F; de Aguiar Vallim, Thomas Q; Shroyer, Noah; Lagor, William R | Arteriosclerosis, thrombosis, and vascular biology | 35172604 | 2022 Apr |
| Genome-wide detection of CRISPR editing in vivo using GUIDE-tag. | Liang, Shun-Qing; Liu, Pengpeng; Smith, Jordan L; Mintzer, Esther; Maitland, Stacy; Dong, Xiaolong; Yang, Qiyuan; Lee, Jonathan; Haynes, Cole M; Zhu, Lihua Julie; Watts, Jonathan K; Sontheimer, Erik J; Wolfe, Scot A; Xue, Wen | Nature communications | 35064134 | 2022 Jan 21 |
| Tetrazine-Ligated CRISPR sgRNAs for Efficient Genome Editing. | Chen, Zexiang; Devi, Gitali; Arif, Amena; Zamore, Phillip D; Sontheimer, Erik J; Watts, Jonathan K | ACS chemical biology | 35446558 | 2022 May 20 |
| Nanoparticle single-cell multiomic readouts reveal that cell heterogeneity influences lipid nanoparticle-mediated messenger RNA delivery. | Dobrowolski, Curtis; Paunovska, Kalina; Schrader Echeverri, Elisa; Loughrey, David; Da Silva Sanchez, Alejandro J; Ni, Huanzhen; Hatit, Marine Z C; Lokugamage, Melissa P; Kuzminich, Yanina; Peck, Hannah E; Santangelo, Philip J; Dahlman, James E | Nature nanotechnology | 35768613 | 2022 Aug |
| Piperazine-derived lipid nanoparticles deliver mRNA to immune cells in vivo. | Ni, Huanzhen; Hatit, Marine Z C; Zhao, Kun; Loughrey, David; Lokugamage, Melissa P; Peck, Hannah E; Cid, Ada Del; Muralidharan, Abinaya; Kim, YongTae; Santangelo, Philip J; Dahlman, James E | Nature communications | 35970837 | 2022 Aug 15 |
| Synthesis and ex vivo biodistribution of two 68Ga-labeled tetrazine tracers: Comparison of pharmacokinetics. | Lambidis, Elisavet; Lumén, Dave; Koskipahta, Elina; Imlimthan, Surachet; Lopez, Brianda B; Sánchez, Ana Isabel Fraguas; Sarparanta, Mirkka; Cheng, R Holland; Airaksinen, Anu J | Nuclear medicine and biology | 35680503 | 2022 |
| Surface conjugation of antibodies improves nanoparticle uptake in bronchial epithelial cells. | Luks, Valerie L; Mandl, Hanna; DiRito, Jenna; Barone, Christina; Freedman-Weiss, Mollie R; Ricciardi, Adele S; Tietjen, Gregory G; Egan, Marie E; Saltzman, W Mark; Stitelman, David H | PloS one | 35385514 | 2022 |
| Antispacer peptide nucleic acids for sequence-specific CRISPR-Cas9 modulation. | Economos, Nicholas G; Quijano, Elias; Carufe, Kelly E W; Perera, J Dinithi R; Glazer, Peter M | Nucleic acids research | 35235944 | 2022 Jun 10 |
| An ELISA-based platform for rapid identification of structure-dependent nucleic acid-protein interactions detects novel DNA triplex interactors. | Economos, Nicholas G; Thapar, Upasna; Balasubramanian, Nanda; Karras, Georgios I; Glazer, Peter M | The Journal of biological chemistry | 35988651 | 2022 Oct |
| 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 |
| The balance between protective and pathogenic immune responses to pneumonia in the neonatal lung is enforced by gut microbiota. | Stevens, Joseph; Steinmeyer, Shelby; Bonfield, Madeline; Peterson, Laura; Wang, Timothy; Gray, Jerilyn; Lewkowich, Ian; Xu, Yan; Du, Yina; Guo, Minzhe; Wynn, James L; Zacharias, William; Salomonis, Nathan; Miller, Lisa; Chougnet, Claire; O'Connor, Dennis Hartigan; Deshmukh, Hitesh | Science translational medicine | 35704600 | 2022 Jun 15 |
| AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice. | Wang, Jiaming; Zhang, Yue; Mendonca, Craig A; Yukselen, Onur; Muneeruddin, Khaja; Ren, Lingzhi; Liang, Jialing; Zhou, Chen; Xie, Jun; Li, Jia; Jiang, Zhong; Kucukural, Alper; Shaffer, Scott A; Gao, Guangping; Wang, Dan | Nature | 35322228 | 2022 Apr |
| Material Engineering in Gut Microbiome and Human Health. | Yang, Letao; Hung, Lin Y; Zhu, Yuefei; Ding, Suwan; Margolis, Kara G; Leong, Kam W | Research (Washington, D.C.) | 35958108 | 2022 |
| 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 |
| rAAV-delivered PTEN therapeutics for prostate cancer. | Ai, Jianzhong; Li, Jia; Su, Qin; Ma, Hong; Wei, Qiang; Li, Hong; Gao, Guangping | Molecular therapy. Nucleic acids | 34976432 | 2022 Mar 08 |
| In Vivo Gene Editing in Lipid and Atherosclerosis Research. | De Giorgi, Marco; Jarrett, Kelsey E; de Aguiar Vallim, Thomas Q; Lagor, William R | Methods in molecular biology (Clifton, N.J.) | 35237996 | 2022 |
| Identification and Validation of CRISPR/Cas9 Off-Target Activity in Hematopoietic Stem and Progenitor Cells. | Park, So Hyun; Lee, Ciaran M; Bao, Gang | Methods in molecular biology (Clifton, N.J.) | 35507169 | 2022 |
| A High-Efficiency AAV for Endothelial Cell Transduction Throughout the Central Nervous System. | Krolak, Trevor; Chan, Ken Y; Kaplan, Luke; Huang, Qin; Wu, Jason; Zheng, Qingxia; Kozareva, Velina; Beddow, Thomas; Tobey, Isabelle G; Pacouret, Simon; Chen, Albert T; Chan, Yujia A; Ryvkin, Daniel; Gu, Chenghua; Deverman, Benjamin E | Nature cardiovascular research | 35571675 | 2022 Apr |
| 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 |
| Gene editing in a Myo6 semi-dominant mouse model rescues auditory function. | Xue, Yuanyuan; Hu, Xinde; Wang, Daqi; Li, Di; Li, Yige; Wang, Fang; Huang, Mingqian; Gu, Xi; Xu, Zhijiao; Zhou, Jinan; Wang, Jinghan; Chai, Renjie; Shen, Jun; Chen, Zheng-Yi; Li, Geng-Lin; Yang, Hui; Li, Huawei; Zuo, Erwei; Shu, Yilai | Molecular therapy : the journal of the American Society of Gene Therapy | 34174443 | 2022 Jan 05 |
| Lung-selective mRNA delivery of synthetic lipid nanoparticles for the treatment of pulmonary lymphangioleiomyomatosis. | Qiu, Min; Tang, Yan; Chen, Jinjin; Muriph, Rachel; Ye, Zhongfeng; Huang, Changfeng; Evans, Jason; Henske, Elizabeth P; Xu, Qiaobing | Proceedings of the National Academy of Sciences of the United States of America | 35173043 | 2022 Feb 22 |
| In Vivo MRI Tracking of Tumor Vaccination and Antigen Presentation by Dendritic Cells. | Bulte, Jeff W M; Shakeri-Zadeh, Ali | Molecular imaging and biology | 34581954 | 2022 Apr |
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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 |
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| Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy. | Pickar-Oliver, Adrian; Gough, Veronica; Bohning, Joel D; Liu, Siyan; Robinson-Hamm, Jacqueline N; Daniels, Heather; Majoros, William H; Devlin, Garth; Asokan, Aravind; Gersbach, Charles A | Molecular therapy : the journal of the American Society of Gene Therapy | 34509668 | 2021 Nov 03 |
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