CRISPR/Cas9 Technology Research Grant - UT Southwestern Medical Center

CRISPR/Cas9 Technology Research Grant - UT Southwestern Medical Center

Funder: Parent Project Muscular Dystrophy (PPMD)

Award Amount: $250,000

Focus Area: Gene therapy and CRISPR/Cas9 technology research for Duchenne muscular dystrophy

Award Date: January 30, 2017

Background

This grant is part of PPMD's Gene Transfer Initiative, which supports early-stage, innovative research at critical moments in a therapy's development. Gene transfer has been explored for years as a possible therapeutic approach to treating Duchenne. CRISPR/Cas9 has recently emerged as a potential therapy with the capability to permanently alter DNA.

Project Description

The grant supports Dr. Eric Olson and the Department of Molecular Biology at UT Southwestern Medical Center's ongoing study of CRISPR/Cas9 technology as a potential treatment for Duchenne.

Research Scope:

Study of various types of dystrophin proteins produced using CRISPR/Cas9 in mouse models

Comparison of dystrophin proteins produced from CRISPR/Cas9 to proteins produced from other therapeutic approaches (e.g., microdystrophins researched for gene transfer)

Investigation of "off-target" effects - potential unintended cuts in genetic code when the CRISPR RNA guidewires target areas close but not exact to intended locations

Comprehensive examination of safety, including potential immune responses to AAV9

Rationale

PPMD believes there is no single magic wand to stop Duchenne progression. A combination of therapies will be needed, and CRISPR/Cas9 with its potential to permanently alter DNA could be an important part of the solution.

Funding Sources

This grant was made possible through:
• Individual donations from the Duchenne community
• Matching gift from the Killian family
• Contributions from Duchenne foundations: The Ackerman Foundation and Nicholoff Family, Hope for Gabe, Hope for Gus, John Owen's Adventure, Kindness Over Muscular Dystrophy (including Curran & Leahy families), Little Hercules Foundation, Ryan's Hope for a Cure Charitable Foundation, and the Zack Heger Foundation
• PPMD's 2016 year-end campaign

Geographic Scope

United States (UT Southwestern Medical Center, Dallas, TX)

Eligibility

Competitive grant awarded to academic medical centers and their research teams

Contact Information

• Phone: 1-800-714-5437
• Email: info@parentprojectmd.org
• Address: 1012 14th Street, NW, Suite 500, Washington, DC 20005
• Website: parentprojectmd.org