Project Grant P50HD117373

Award Date 8/15/24
Completion Date 7/31/28
Dollars Obligated $1.6M
Federal Grant Program
93.865
Assistance Type
Project Grant
Place of Performance
Columbus, OH 43205, USA
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This $1,557,819 Project Grant award from the National Institute of Child Health and Human Development (CFDA 93.865 - Child Health and Human Development Extramural Research) supports muscular dystrophy research at the Research Institute at Nationwide Children's Hospital in Columbus, Ohio. The award funds three key research projects:

  1. Project 1 (PI: Paul Martin, PhD) aims to utilize a novel bicistronic AAV vector to both prevent muscle disease and build new muscle function in patients with limb girdle muscular dystrophy 2I.

  2. Project 2 (PI: Kevin Flanigan, MD) will develop vectors containing U7 small nuclear RNAs to restore functional dystrophin expression.

  3. Project 3 (PI: Nizar Saad, PhD) seeks to identify novel circulating biomarkers and develop improved gene therapy approaches to increase transduction efficiency, dystrophin expression, and reduce immune responses.

The award also supports a Muscular Dystrophy Cell and Serum Banking Core, a Training Core to expand educational offerings, and an Administrative Core to facilitate community outreach. This five-year grant, awarded on August 15, 2024, leverages the institute's expertise in gene therapy and muscular dystrophy research to advance new treatment paradigms.

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