MyFSHD
MyFSHD
Peter L Jones, PhD
The nuts and bolts of gene therapy for FSHD (Part 1 of 2)
1 hour 19 minutes Posted Aug 15, 2025 at 7:23 pm.
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"My friends are toys. I make them. It's a hobby. I'm a genetic designer." This is a two part podcast covering the important factors is successful gene therapy and introduces the knowledge gained from other neuromuscular diseases and how it can be used to positively impact gene therapy in FSHD. In part 1, we focus on AAV serotypes, transgene constructs, immune responses, and delivery. We look into DMD gene therapy and how that can impact the current FSHD gene therapy programs. Check us out on the MyFSHD Youtube channel as well.