Scientific Research Resource Core

NIH RePORTER · NIH · P50 · $199,454 · view on reporter.nih.gov ↗

Abstract

Project Summary The breakthrough technology of CRISPR/Cas9-mediated genomic editing offers an entirely new and powerful therapeutic potential for patients suffering from genetic diseases like Duchenne Muscular Dystrophy (DMD). Since joining the Wellstone Network in 2015, the UT Southwestern Myoediting Core has served both as an essential coordinating hub for the Wellstone research groups (Projects 1 & 2) at UT Southwestern and as a conduit for sharing expertise and resources with the broader scientific community. Moving forward, our goals for this Shared Research Resource Core are: (Aim #1) To continue to support Projects 1 and 2 of the UT Southwestern Wellstone Center by providing the cutting-edge technologies of human iPSCs, CRISPR/Cas9 genomic editing, and cardiomyocyte differentiation. (Aim #2) To help advance the study of muscular dystrophy worldwide by providing a DMD/BMD Biorepository of patient-derived DMD iPSCs and their corresponding CRISPR/Cas9-corrected isolates along with information regarding the clinical phenotype and genomic sequence of the donor patient. (Aim #3) To help other investigators take full advantage of these resources by freely sharing our streamlined protocols and providing training (either on site, or in silico) in best practices and approaches. These resources are available to investigators from other Wellstone Centers as well as the general scientific community at large.

Key facts

NIH application ID
10049127
Project number
2P50HD087351-06
Recipient
UT SOUTHWESTERN MEDICAL CENTER
Principal Investigator
RHONDA BASSEL-DUBY
Activity code
P50
Funding institute
NIH
Fiscal year
2020
Award amount
$199,454
Award type
2
Project period
2015-09-15 → 2025-08-31