Evaluation of safety and early efficacy of AAV gene therapy in mouse models of vanishing white matter diseaseJessica A. Herstine(Nationwide Children's Hospital), Allison M. Bradbury(Nationwide Children's Hospital)Molecular TherapyMarch 27, 202410.1016/j.ymthe.2024.03.034Cited by 9SaveCiteExport RISWatch citationsRelated PapersDirect Intracranial Injection of AAVrh8 Encoding Monkey β-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain|Human Gene Therapy|2017|93AAV Gene Therapy Strategies for Lysosomal Storage Disorders with Central Nervous System Involvement|Neuromethods|2015|6