Direct Intracranial Injection of AAVrh8 Encoding Monkey β-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate BrainDiane Golebiowski, Miguel Sena‐Esteves, Nina Bishop et al.|Human Gene Therapy|2017Cited by 93
Pronounced Therapeutic Benefit of a Single Bidirectional AAV Vector Administered Systemically in Sandhoff MiceHannah G. Lahey, Miguel Sena‐Esteves, Chelsea Joy Webber et al.|Molecular Therapy|2020Cited by 36
AAV Gene Therapy Strategies for Lysosomal Storage Disorders with Central Nervous System InvolvementDiane Golebiowski, Miguel Sena‐Esteves, Allison M. Bradbury et al.|Neuromethods|2015Cited by 6
710. Optimization of AAV Vector Design for Safe Expression of β-N-Acetylhexosaminidase in the Brain for Tay-Sachs Disease Gene TherapyDiane Golebiowski, Miguel Sena‐Esteves, Keiko Y. Petrosky et al.|Molecular Therapy|2015Cited by 0