Adeno-associated virus (AAV) mediated gene therapy continues to be a promising therapeutic path for various diseases, including monogenic neuromuscular and cardiac indications such as Duchenne muscular dystrophy (DMD) and Friedreich’s ataxia (FA). However, the high systemic doses currently required to achieve widespread therapeutic benefit can pose potential safety risks. These risks could be decreased or eliminated if therapeuticbenefit could be achieved using lower doses through a more targeted and efficacious vector.
Wednesday, May 17, 2023
Novel AAV Capsid Identification and Characterization for Neuromuscular and Cardiac Indications
1549 Novel AAV Capsid Identification and Characterization for Neuromuscular and Cardiac Indications; Jennifer C. G. Green, Jessica F. Boehler, Meghan S. Soustek, Jamie L. Marshall, Tiffany Willacy, Prushti Bhavsar, Kristy J. Brown, Sharon McGonigle, Carl Morris (Solid Biosciences). May 01, 2023 Volume 31Issue 4Supplement 1S1-794 26th Annual Meeting of the American Society of Gene & Cell Therapy.
