AAV9 Gene Therapy Optimization for SMARD1/CMT2S: Safety and Long-Term Efficacy Comparison of Two Vectors in a SMARD1 Preclinical Model

Investigators compared the efficacy of two novel, optimized AAV9-Immunoglobulin Mu DNA Binding Protein 2 vectors, utilizing either the chicken β-actin or a truncated form of the methyl-CpG-binding protein 2 promoter.