NIH R01 · 2025
Allele Specific Knockdown for LGMDD1
PROJECT SUMMARY / ABSTRACT While the promise of gene-based therapies for disabling neuromuscular diseases is finally becoming a reality, research efforts thus far have primarily focused on gene replacement strategies for recessive, loss-of-function disorders. Such strategies are not translatable to most dominant muscular dystrophies, hindering the development of new treatment strategies.1,2 Our group recently identified mutations in DNAJB6 that cause limb girdle muscular dystrophy D1 (LGMDD1), a dominantly inherited disabling myopathy with no current treatment options.3 The overarching goal of this proposal is to develop novel therapies for this devastating disease. Addressing this unmet…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.