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 |