Ana Rita Batista

Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne’s Muscular Dystrophy

Angela Lek, Brenda Wong, Allison M. Keeler et al. · New England Journal of Medicine · 2023 · 273 citations · Full text

AAV gene therapy for Tay-Sachs disease

Terence R. Flotte, Oğuz Çataltepe, Ajit S Puri et al. · Nature Medicine · 2022 · 122 citations · Full text

Gene Therapies, Aav Gene Therapy, Pathogenesis +5

A Safe and Reliable Technique for CNS Delivery of AAV Vectors in the Cisterna Magna

Toloo Taghian, Miklós Marosföi, Ajit S Puri et al. · Molecular Therapy · 2019 · 102 citations · Full text

<i>Ly6a</i> Differential Expression in Blood–Brain Barrier Is Responsible for Strain Specific Central Nervous System Transduction Profile of AAV-PHP.B

Ana Rita Batista, Oliver D. King, Christopher Reardon et al. · Human Gene Therapy · 2019 · 100 citations

Anti-apoptotic treatment reduces transthyretin deposition in a transgenic mouse model of Familial Amyloidotic Polyneuropathy

Bárbara Macedo, Ana Rita Batista, Nelson Ferreira et al. · Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease · 2008 · 66 citations · Full text

Neurodegenerative Diseases, Alzheimer's Disease, Familial Amyloidotic Polyneuropathy +8