Project Grant R01HL169793

Award Date 8/15/24
Completion Date 5/31/28
Dollars Obligated $758K
Federal Grant Program
93.837
Assistance Type
Project Grant
Place of Performance
Seattle, WA 98101, USA
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This federal Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI), under the Cardiovascular Diseases Research program (CFDA 93.837), aims to develop a novel gene editing therapy for the treatment of hemophilia A. The $758,254 award will fund research to combine lipid nanoparticles and CRISPR/Cas9 gene editing tools to correct the mutant factor VIII gene and restore normal blood clotting in hemophilia patients.

The project will first investigate the correction of small gene deletions/insertions in a hemophilia mouse model using the optimized lipid nanoparticle delivery system. It will then explore the use of base editing technology to correct single base mutations in patient-derived blood cells, with the goal of developing personalized gene editing approaches for hemophilia A. The research will be conducted primarily by Seattle Children's Hospital, with a sub-award to the University of Washington to provide hemophilia clinical expertise and patient samples for the study.

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