Originally presented on September 10th, 2021 as a part of the Professional Track at the 2021 Virtual MDF Annual Conference.
Repeat instability is the crux of myotonic dystrophy disease. Join three leading researchers for a panel presentation of research addressing possible opportunities for repeat stabilization. Dr. Pearson presents recent research into mouse model repeat stabilization for Huntington's with a discussion of implications for DM. Dr. Monckton discusses somatic CTG repeat expansion in DM1, with respect to dynamics, consequences, and therapeutic opportunities. Dr. Bialek discusses the work of Triplet Therapeutics in utilizing ASOs and siRNAs to stabilize repeat expansion.
- Peter Bialek, PhD
- Darren Monckton, PhD
- Christopher Pearson, PhD