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
| 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
Genome Editor Projects
| 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
| 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.
| Year | Title | Collaborators | Collaboration Institution |
|---|---|---|---|
| 2019-2020 | Evaluation of LNP-based nanoparticles for systematic delivery of base editors | Peter Glazer, MD PhD | Yale University |
| W. Mark Saltzman, PhD | Yale University | ||
| Alice Tarantal, PhD | University of California at Davis | ||
| 2019-2020 | Improved reporters to evaluate base editor activity in mice | Steve Murray, PhD | Jackson Laboratory |
| David Liu, PhD | Broad Institute | ||
| 2019-2020 | Development of a GFP-IVS2 Mouse Reporter Strain for PNA-based Genome Editing Detection | Steve Murray, PhD | Jackson Laboratory |
| Peter Glazer, MD PhD | Yale University | ||
| 2019-2020 | Nanoparticles delivery of new Cas enzymes | Niren Murthy, PhD | University of California Berkeley |
| Jennifer Doudna, PhD | University of California Berkeley | ||
| 2019-2020 | Comparing the efficiency and cellular impact of non-viral, hepatocyte-targeted delivery vehicles with viral-mediated gene delivery in a microphysiological liver on chip platform | Samira Kiani, PhD | University of Pittsburgh |
| Shaoqin Gong, PhD | University of Wisconsin-Madison | ||
| Ross Wilson, PhD | University of California Berkeley | ||
| 2019-2020 | Non-enzymatic gene editing of hematopoietic cells in utero in nonhuman primates by next generation PNA nanoparticles | Zheng-Yi Chen, PhD | Massachusetts Eye and Ear Infirmary |
| Qiaobing Xu, PhD | Tufts University | ||
| David Liu, PhD | Broad Institute | ||
| 2019-2020 | Adenine Base Editor Ribonucleoprotein Delivery to Primary Airway Epithelia using Amphiphilic Peptids | Paul McCray, MD | University of Iowa |
| Alice Tarantal, PhD | University of California at Davis | ||
| David Liu, PhD | Broad Institute | ||
| David Guay, PhD | Feldan Institute | ||
| 2019-2020 | RNP delivery platforms for biodistribution of genome editors to human neural microtissues | Todd McDevitt, PhD | Gladstone Institute |
| Ross Wilson, PhD | University of California Berkeley | ||
| 2020-2021 | Guide Engineering to Enhance the Safety and Efficacy of Base Editing in vivo | Erik Sontheimer, PhD | University of Massachusetts Medical School |
| David Liu, PhD | Broad Institute | ||
| 2020-2021 | Development of a single AAV vector CRISPR-CasΦ platform technology | Jennifer Doudna, PhD | University of California Berkeley |
| David Liu, PhD | Broad Institute | ||
| Ben Deverman, PhD | Broad Institute | ||
| 2020-2021 | Novel virus-like particles (VLPs) for precise delivery of base-editors in iPSC disease models | Bruce Conklin, MD | Gladstone Institute |
| Jennifer Doudna, PhD | University of California Berkeley | ||
| 2020-2021 | Cas9 ribonucleoprotein delivery targeted to kidney epithelium | Ross Wilson, PhD | University of California Berkeley |
| Benjamin Freedman, PhD | University of Washington | ||
| 2020-2021 | Developing methods for unbiased off-target genome editing analysis in vivo | Scot Wolfe, PhD | University of Massachusetts Medical School |
| Wen Xue, PhD | University of Massachusetts Medical School | ||
| 2020-2021 | Improving Split AAV9 Base Editors In Mice | Cathleen Lutz, PhD | Jackson Laboratory |
| David Liu, PhD | Broad Institute | ||
| 2020-2021 | CEST based imaging of Cas9-b-lactamase fusion proteins | Niren Murthy, PhD | University of California Berkeley |
| Moriel Vandsburger, PhD | University of California Berkeley | ||
| 2021-2022 | Assessment of Prime Editing in Rhesus Monkeys | David Liu, PhD | Broad Institute |
| Alice Tarantal, PhD | University of California at Davis | ||
| 2021-2022 | Enhanced in vivo editing of the endothelium via a novel adenovirus-AAV chimera vector | David Curiel, MD PhD | Washington University in St. Louis |
| Gang Bao, PhD | Rice University | ||
| Bill Lagor, PhD | Baylor College of Medicine | ||
| 2021-2022 | Identification 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 pig | Zheng-Yi Chen, PhD | Massachusetts Eye and Ear Infirmary |
| David Guay, PhD | Feldan Institute | ||
| 2021-2022 | Nanoparticle-based delivery of Mitochondrial Based Editors | Steve Ekker, PhD | Mayo Clinic |
| Sarah Gong, PhD | University of Wisconsin-Madison | ||
| 2021-2022 | Libraries of Cas9-mRNA nanoparticles in human iPSC-derived neurons | Bruce Conklin, MD | Gladstone Institute |
| James Dahlman, PhD | Georgia Institute of Technology | ||
| 2021-2022 | Editing the Kidney Epigenome | Beno Freedman, PhD | University of Washington |
| Charlie Gersbach, PhD | Duke University | ||
| Aravind Asokan, PhD | Duke University | ||
| 2021-2022 | Evaluation of Immune Responses to Novel Editors in Rhesus Monkeys | Jennifer Doudna, PhD | University of California Berkeley |
| Alice Tarantal, PhD | University of California at Davis |