This dystrophin gene correction approach which involves editing muscle satellite stem cells potentially enhances durability and functional outcomes compared to synthetic approaches. Since up to 60% of DMD cases are caused by defects between exons 45 and 55, this approach is more broadly applicable for the majority of DMD patients than exon skippers.
Key findings from the study include:
- Functional dystrophin protein production: PBGENE-DMD restored the body’s ability to produce a functional dystrophin protein across multiple muscles, including heart, diaphragm, and skeletal muscles at levels expected to provide therapeutic benefit.
- Enhanced Muscle Resilience: Treated mice exhibited a 66% improvement in resistance to eccentric injury, an indicator of enhanced muscle resilience, compared to untreated diseased counterparts.
- Long-Term Functional Improvement: In mice treated with PBGENE-DMD the maximum force output (MFO), a critical functional metric, reached up to 93% of the MFO in healthy control mice with improvement observed in PBGENE-DMD-treated mice between 3 and 6 months.
- Durable Outcomes: PBGENE-DMD-edited dystrophin mRNA transcript was detected in PAX7+ cells, a marker for muscle satellite stem cells, suggesting potential for durable therapeutic effects compared to standard gene therapy approaches.
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