Funded Research

Innovative Technologies to Non-Invasively Monitor Genome Edited Cells In Vivo (UH2/UH3 Clinical Trial Not Allowed) RFA-RM-18-025
PI Name Institution Name Title
BULTE, JEFF W JOHNS HOPKINS UNIVERSITY Non-Invasive Tracking of Genome-Corrected iPS cells in ALS
RONALD, JOHN ANDREW UNIVERSITY OF WESTERN ONTARIO Non-Invasive Monitoring of CRISPR/Cas-Edited Chimeric Antigen Receptor T (CAR-T) Cells with Reporter Gene-Based Magnetic Resonance Imaging and Positron Emission Tomography
TARANTAL, ALICE F (contact)
SEGAL, DAVID J
UNIVERSITY OF CALIFORNIA AT DAVIS Innovative Translational Imaging Technologies to Monitor Genome Edited Cells in Vivo
VANDSBURGER, MORIEL UNIVERSITY OF CALIFORNIA BERKELEY Molecular MRI for in vivo tracking of gene editing and gene edited cells

 

Expanding the Human Genome Engineering Repertoire (U01 Clinical Trial Not Allowed) RFA-RM-18-024
PI Name Institution Name Title
GERSBACH, CHARLES A DUKE UNIVERSITY Epigenome Editing Technologies for Treating Diverse Disease
GLAZER, PETER M (contact)
LY, DANITH H
SALTZMAN, W. MARK
YALE UNIVERSITY PNA Nanoparticles for Gene Editing In Vivo

 

Innovative Technologies to Deliver Genome Editing Machinery to Disease-relevant Cells and Tissues (UG3/UH3 Clinical Trial Not Allowed) RFA-RM-18-023 
PI Name Institution Name Title
BANKIEWICZ, KRYSTOF S (contact)
MURTHY, NIREN
OHIO STATE UNIVERSITY Development of a nanoparticle-based gene editing technology for neurological applications
BAO, GANG (contact)
LAGOR, WILLIAM RAYMOND
SUH, JUNGHAE
RICE UNIVERSITY Velcro AAV Vector for tissue-specific delivery of genome editing reagents with enhanced cargo capacity
CHAIKOF, ELLIOT BETH ISRAEL DEACONESS MEDICAL CENTER Delivery Technologies for In Vivo Genome Editing
CURIEL, DAVID TERRY WASHINGTON UNIVERSITY Endothelial-targeted adenovirus for organ-selective gene editing in vivo
DAHLMAN, JAMES (contact)
SANTANGELO, PHILIP J
GEORGIA INSTITUTE OF TECHNOLOGY Highly Specific ZFN-Based HSC Gene Editing Therapies Identified By In Vivo Barcode Nanoparticle Screens And Rationally Designed Mrna
LAM, KIT S (contact)
CHENG, R.HOLLAND
UNIVERSITY OF CALIFORNIA AT DAVIS Cell-specific nanocarrier with endocytic and endosomolytic activities for therapeutic genome editing
LEONG, KAM W COLUMBIA UNIVERSITY HEALTH SCIENCES Focused Ultrasound-mediated Delivery of Gene-editing Elements to the Brain for Neurodegenerative Disorders
TILTON, JOHN CHRISTIAN CASE WESTERN RESERVE UNIVERSITY In vivo delivery of CRISPR Cas9-guide RNA nucleoprotein complexes using the nanoPOD platform
WILSON, ROSS (contact)
DOUDNA, JENNIFER A
UNIVERSITY OF CALIFORNIA BERKELEY Cas9 RNP delivery to immune cells in vivo via molecular targeting
YI, GUOHUA TEXAS TECH UNIVERSITY HEALTH SCIS CENTER Novel CRISPR-Cas9 protein delivery to T cells in vivo by targeting CD7
ZHOU, JIANGBING YALE UNIVERSITY Novel grafted terpolymers for targeted delivery of CRISPR/Cas9- mediated precise genome editing to the brain

 

Development of Cell and Tissue Platforms to Detect Adverse Biological Consequences of Somatic Cell Genome Editing (U01 Clinical Trial Not Allowed) RFA-RM-18-022
PI Name Institution Name Title
FREEDMAN, BENJAMIN SOLOMON UNIVERSITY OF WASHINGTON Improving the Safety of Genome Editing With Human Kidney Organoids
GERSBACH, CHARLES A. (contact)
BURSAC, NENAD
TRUSKEY, GEORGE A
DUKE UNIVERSITY Microphysiological Human Tissue Systems for Monitoring of Genome Editing Outcomes
HINSON, JOHN TRAVIS UNIVERSITY OF CONNECTICUT SCH OF MED/DNT Human cardiac microtissues with innate immune sensing to study adverse consequences of genome editing
MORIZANE, RYUJI (contact)
LEWIS, JENNIFER A.
SABBISETTI, VENKATA
MASSACHUSETTS GENERAL HOSPITAL Vascularized kidney organoids on chip for efficacy and toxicity testing of somatic genome editing

 

Large Animal Testing Centers for Evaluation of Somatic Cell Genome Editing Tools (U42 Clinical Trial Not Allowed) RFA-RM-18-014
PI Name Institution Name Title
TARANTAL, ALICE F (contact) 
SEGAL, DAVID J 
UNIVERSITY OF CALIFORNIA AT DAVIS Nonhuman Primate Testing Center for Evaluation of Somatic Cell Genome Editing Tools
WELLS, KEVIN DALE (contact) 
PRATHER, RANDALL S
UNIVERSITY OF MISSOURI-COLUMBIA Swine Somatic Cell Genome Editing (SCGE) Center

 

 

Somatic Cell Genome Editing Dissemination and Coordination Center (U24 - Clinical Trial Not Allowed) RFA-RM-18-018
PI Name Institution Name Title
DWINELL, MELINDA R (contact) 
SHIMOYAMA, MARY E 
MEDICAL COLLEGE OF WISCONSIN Dissemination and Coordinating Center for the SCGE Consortium

 

Rodent Testing Centers for Development of Reporter Systems and Evaluation of Somatic Cell Genome Editing Tools (U42 Clinical Trial Not Allowed) RFA-RM-18-012
PI Name Institution Name Title
HEANEY, JASON D (contact) 
DICKINSON, MARY E 
LAGOR, WILLIAM RAYMOND
BAYLOR COLLEGE OF MEDICINE BCM-Rice resource for the analysis of somatic gene editing in mice
MURRAY, STEPHEN A (contact) 
LUTZ, CATHLEEN M
JACKSON LABORATORY The Jackson Laboratory Gene Editing Testing Center (JAX-GETC)

 

Development of Large Animal Reporter Systems for Testing Somatic Cell Genome Editing Tools (U24 Clinical Trial Not Allowed) RFA-RM-18-013
PI Name Institution Name Title
CARLSON, DANIEL FRED RECOMBINETICS, INC. Development of Swine Reporter Models for Testing Somatic Cell Genome Editing Tools
FENG, GUOPING MASSACHUSETTS INSTITUTE OF TECHNOLOGY Knockin marmoset reporters for non-invasive measuring of genome-editing efficiency
HENNEBOLD, JON D  OREGON HEALTH & SCIENCE UNIVERSITY Rhesus Macaque Somatic Cell Gene Editing Resource

 

Development of Cell and Tissue Platforms to Detect Adverse Biological Consequences of Somatic Cell Gene Editing (U01 Clinical Trial Not Allowed) RFA-RM-18-015
PI Name Institution Name Title
KIANI, SAMIRA ARIZONA STATE UNIVERSITY-TEMPE CAMPUS Multicell type human liver on chip microphysiological platform to examine CRISPR- based gene modulation.
MCDEVITT, TODD C  J. DAVID GLADSTONE INSTITUTES Human microtissues for in situ detection and functional measurement of adverse consequences caused by genome editing
SAHA, KRISHANU (contact) 
GAMM, DAVID M 
ROY, SUSHMITA 
SKALA, MELISSA CAROLINE 
UNIVERSITY OF WISCONSIN-MADISON Single Cell Profiling To Define Biomarkers Of Photoreceptor Dysfunction After Gene Editing Within PSC-Derived Organoids
TSAI, SHENGDAR  ST. JUDE CHILDREN'S RESEARCH HOSPITAL A novel human T-cell platform to define biological effects of genome editing

 

Innovative Technologies to Deliver Genome Editing Machinery to Disease-relevant Cells and Tissues (UG3/UH3 Clinical Trial Not Allowed) RFA-RM-18-016
PI Name Institution Name Title
ASOKAN, ARAVIND DUKE UNIVERSITY Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
CHEN, ZHENG-YI (contact) 
LIU, DAVID R 
XU, QIAOBING
MASSACHUSETTS EYE AND EAR INFIRMARY Efficient in Vivo RNP-based Gene Editing in the Sensory Organ Inner Ear Using Bioreducible Lipid Nanoparticles
DEVERMAN, BENJAMIN E BROAD INSTITUTE, INC. Novel AAVs engineered for efficient and noninvasive cross-species gene editing throughout the central nervous system
GAO, GUANG-PING (contact) 
ANDERSON, DANIEL G 
XUE, WEN 
UNIV OF MASSACHUSETTS MED SCH WORCESTER Develop combinatorial non-viral and viral CRISPR delivery for lung diseases
GHIRAN, IONITA CALIN  BETH ISRAEL DEACONESS MEDICAL CENTER Bioengineered red blood cells as extracellular vesicle-mediated delivery platforms for gene editing machinery
GONG, SHAOQIN (contact) 
EMBORG, MARINA 
LEVINE, JON E 
ROY, SUBHOJIT 
SAHA, KRISHANU
UNIVERSITY OF WISCONSIN-MADISON Enabling Nanoplatforms for Targeted in vivo Delivery of CRISPR/Cas9 Ribonucleoproteins in the Brain
MCCRAY, PAUL B UNIVERSITY OF IOWA Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides
SALTZMAN, W. MARK (contact) 
GLAZER, PETER M 
YALE UNIVERSITY Poly(amine-co-ester)s for Targeted Delivery In Vivo of Gene Editing Agents to Bone Marrow and Lung
SONTHEIMER, ERIK J (contact) 
KHVOROVA, ANASTASIA 
WATTS, JONATHAN K 
WOLFE, SCOT A 
UNIV OF MASSACHUSETTS MED SCH WORCESTER Enhancing CRISPR Gene Editing in Somatic Tissues by Chemical Modification of Guides and Donors

 

Expanding the Human Genome Engineering Repertoire (U01 Clinical Trial Not Allowed) RFA-RM-18-017
PI Name Institution Name Title
DOUDNA, JENNIFER A (contact) 
BANFIELD, JILLIAN 
UNIVERSITY OF CALIFORNIA BERKELEY Expanding CRISPR-Cas editing technology through exploration of novel Cas proteins and DNA repair systems
EKKER, STEPHEN CARL (contact) 
CLARK, KARL J
MAYO CLINIC ROCHESTER Building the mitochondrial genome editing repertoire
LIU, DAVID R BROAD INSTITUTE, INC. Expanding the Scope of Base Editing

 

This page last reviewed on October 2, 2019