Phase 1 of the SCGE program had five different initiatives that project groups worked towards. In addition to the five initiatives, there was a Collaboration Opportunity Fund to promote the exchange, cross-testing, and evaluation of the improved technologies within the consortium. Learn more about the different projects below, and find the latest publications from Phase 1 projects at the link.

Animal Reporter and Testing Centers

  • The initiative goal is to create and use new animal (mouse, pig, marmoset and rhesus macaque) reporter models to accelerate the translation of genome editing technologies into treatments for human diseases. The animal model systems are based on normal, non-diseased animals.

Biological Effects Projects

Delivery Systems Projects

  • The initiative goal is to support the development and evaluation of innovative approaches to deliver genome editing machinery into somatic cells, with the ultimate goal of accelerating the development of genome editing therapeutics to treat human disease.  Funded projects are focusing on a wide array of disease-relevant cell types and testing multiple technologies for delivery to these cell types.

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 
UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL 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
UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL WORCESTER Enhancing CRISPR Gene Editing in Somatic Tissues by Chemical Modification of Guides and Donors
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
UNIVERSITY OF CALIFORNIA, SAN FRANCISCO
Development of a nanoparticle-based gene editing technology for neurological applications
BAO, GANG (contact)
LAGOR, WILLIAM RAYMOND
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
EMORY UNIVERSITY 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 UNIVERSITY OF TEXAS HEALTH CENTER AT TYLER 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

Genome Editor Projects

  • This initiative supports the development of innovative genome editing systems with improved specificity, efficiency or functionality over currently available systems, including the identification of complexes with novel enzymatic activities and substrate specificities.

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
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

Dissemination and Coordinating Center

  • The DCC primary goals are to facilitate interactions and communication between consortium initiatives and disseminate the SCGE Toolkit to the research community.

Collaboration Opportunity Fund (COF) Projects

The Collaboration Opportunity Fund (COF) was meant to promote the exchange, cross-testing and evaluation of the improved technologies within the Somatic Cell Genome Editing (SCGE) Consortium. In phase 1, the COF supported new and pilot research projects led by SCGE-supported investigators.

YearTitleCollaboratorsCollaboration Institution
2019-2020Evaluation of LNP-based nanoparticles for systematic delivery of base editorsPeter Glazer, MD PhDYale University
W. Mark Saltzman, PhDYale University
Alice Tarantal, PhDUniversity of California at Davis
2019-2020Improved reporters to evaluate base editor activity in miceSteve Murray, PhDJackson Laboratory
David Liu, PhDBroad Institute
2019-2020Development of a GFP-IVS2 Mouse Reporter Strain for PNA-based Genome Editing DetectionSteve Murray, PhDJackson Laboratory
Peter Glazer, MD PhDYale University
2019-2020Nanoparticles delivery of new Cas enzymesNiren Murthy, PhDUniversity of California Berkeley
Jennifer Doudna, PhDUniversity of California Berkeley
2019-2020Comparing the efficiency and cellular impact of non-viral, hepatocyte-targeted delivery vehicles with viral-mediated gene delivery in a microphysiological liver on chip platformSamira Kiani, PhDUniversity of Pittsburgh
Shaoqin Gong, PhDUniversity of Wisconsin-Madison
Ross Wilson, PhDUniversity of California Berkeley
2019-2020Non-enzymatic gene editing of hematopoietic cells in utero in nonhuman primates by next generation PNA nanoparticlesZheng-Yi Chen, PhDMassachusetts Eye and Ear Infirmary
Qiaobing Xu, PhDTufts University
David Liu, PhDBroad Institute
2019-2020Adenine Base Editor Ribonucleoprotein Delivery to Primary Airway Epithelia using Amphiphilic PeptidsPaul McCray, MDUniversity of Iowa
Alice Tarantal, PhDUniversity of California at Davis
David Liu, PhDBroad Institute
David Guay, PhDFeldan Institute
2019-2020RNP delivery platforms for biodistribution of genome editors to human neural microtissuesTodd McDevitt, PhDGladstone Institute
Ross Wilson, PhDUniversity of California Berkeley
2020-2021Guide Engineering to Enhance the Safety and Efficacy of Base Editing in vivoErik Sontheimer, PhDUniversity of Massachusetts Medical School
David Liu, PhDBroad Institute
2020-2021Development of a single AAV vector CRISPR-CasΦ platform technologyJennifer Doudna, PhDUniversity of California Berkeley
David Liu, PhDBroad Institute
Ben Deverman, PhDBroad Institute
2020-2021Novel virus-like particles (VLPs) for precise delivery of base-editors in iPSC disease modelsBruce Conklin, MDGladstone Institute
Jennifer Doudna, PhDUniversity of California Berkeley
2020-2021Cas9 ribonucleoprotein delivery targeted to kidney epitheliumRoss Wilson, PhDUniversity of California Berkeley
Benjamin Freedman, PhDUniversity of Washington
2020-2021Developing methods for unbiased off-target genome editing analysis in vivoScot Wolfe, PhDUniversity of Massachusetts Medical School
Wen Xue, PhDUniversity of Massachusetts Medical School
2020-2021Improving Split AAV9 Base Editors In MiceCathleen Lutz, PhDJackson Laboratory
David Liu, PhDBroad Institute
2020-2021CEST based imaging of Cas9-b-lactamase fusion proteinsNiren Murthy, PhDUniversity of California Berkeley
Moriel Vandsburger, PhDUniversity of California Berkeley
2021-2022Assessment of Prime Editing in Rhesus MonkeysDavid Liu, PhDBroad Institute
Alice Tarantal, PhDUniversity of California at Davis
2021-2022Enhanced in vivo editing of the endothelium via a novel adenovirus-AAV chimera vectorDavid Curiel, MD PhDWashington University in St. Louis
Gang Bao, PhDRice University
Bill Lagor, PhDBaylor College of Medicine
2021-2022Identification and optimization of shuttle peptides-mediated local RNP delivery of editing complex in the sensory organs of the inner ear and retina in vivo in mice and the pigZheng-Yi Chen, PhDMassachusetts Eye and Ear Infirmary
David Guay, PhDFeldan Institute
2021-2022Nanoparticle-based delivery of Mitochondrial Based EditorsSteve Ekker, PhDMayo Clinic
Sarah Gong, PhDUniversity of Wisconsin-Madison
2021-2022Libraries of Cas9-mRNA nanoparticles in human iPSC-derived neuronsBruce Conklin, MDGladstone Institute
James Dahlman, PhDGeorgia Institute of Technology
2021-2022Editing the Kidney EpigenomeBeno Freedman, PhDUniversity of Washington
Charlie Gersbach, PhDDuke University
Aravind Asokan, PhDDuke University
2021-2022Evaluation of Immune Responses to Novel Editors in Rhesus MonkeysJennifer Doudna, PhDUniversity of California Berkeley
Alice Tarantal, PhDUniversity of California at Davis