Procession to IND of a capsid mutated AAV8 codon optimized NAGLU vector for treatment of Sanfilippo syndrome type B

Molecular Genetics and Metabolism 2026

View at publisher

Authors: Courtney J. Rouse, Fatima Shaerzadeh, Lingbao Ai, Barry Byrne, Coy Don Heldermon

DOI: 10.1016/j.ymgme.2025.109602

Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Virus-based gene therapy research RNA Interference and Gene Delivery