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.

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
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, DAVIS
 Nonhuman Primate Testing Center for Evaluation of Somatic Cell Genome Editing Tools
WELLS, KEVIN
(contact
PRATHER, RANDALL		 UNIVERSITY OF MISSOURI, COLUMBIA Swine Somatic Cell Genome Editing (SCGE) Center

Biological Effects Projects

Biological Systems Projects

The initiative goal is to support the development, validation and testing of new and existing human cell- and tissue-based platforms that can provide information on the safety of genome editing technologies and delivery systems. These models will be used for preclinical testing of editing, delivery and efficacy.

In Vivo Cell Tracking Projects

The objective of the program is to support the development of tools and technologies that will enable longitudinal monitoring and tracking of genome-edited cells in humans to better assess the safety and efficacy of genome editing therapies. This will be accomplished through the development of innovative non-invasive technologies to label and track genome-edited cells in vivo, ideally in a clinically-relevant matter that has the potential to assess long-term safety in genome editing clinical trial participants.

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 UNIVERSITY OF PITTSBURGH AT PITTSBURGH Multicell type human liver on chip microphysiological platform to examine CRISPR- based gene modulation.
CONKLIN, BRUCE  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
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
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

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.

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

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