Award
National Institute of Neurological Disorders and Stroke 7K01NS134784-03
Development of in Vivo Base Editing as a Genetic Treatment for Spinal Muscular Atrophy
Recipient
UNIVERSITY OF PITTSBURGH AT PITTSBURGH
Awarded
May 12, 2026
Identifier
7K01NS134784-03
NIH grant awarded to develop in vivo base editing as a genetic treatment for spinal muscular atrophy, involving advanced genome editing and preclinical studies.
Description
This is a NIH grant project focused on developing gene editing therapies for spinal muscular atrophy (SMA). The project involves advanced genome editing technologies, preclinical studies, and collaboration with NIH study sections. The project aims to create a lasting, single-dose genetic treatment for SMA using base editing and viral delivery methods.