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Sena-Esteves, Miguel
One or more keywords matched the following items that are connected to
Sena-Esteves, Miguel
Item Type
Name
Concept
beta-Hexosaminidase alpha Chain
Concept
beta-Hexosaminidase beta Chain
Concept
beta-N-Acetylhexosaminidases
Academic Article
Direct Intracranial Injection of AAVrh8 Encoding Monkey ?-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain.
Academic Article
Adeno-Associated Virus Gene Therapy in a Sheep Model of Tay-Sachs Disease.
Academic Article
Biomarkers for disease progression and AAV therapeutic efficacy in feline Sandhoff disease.
Academic Article
Mucopolysaccharidosis-like phenotype in feline Sandhoff disease and partial correction after AAV gene therapy.
Academic Article
Therapeutic response in feline sandhoff disease despite immunity to intracranial gene therapy.
Academic Article
Pronounced Therapeutic Benefit of a Single Bidirectional AAV Vector Administered Systemically in Sandhoff Mice.
Academic Article
Therapeutic benefit after intracranial gene therapy delivered during the symptomatic stage in a feline model of Sandhoff disease.
Academic Article
Upregulating ?-hexosaminidase activity in rodents prevents a-synuclein lipid associations and protects dopaminergic neurons from a-synuclein-mediated neurotoxicity.
Academic Article
Cerebral organoids derived from Sandhoff disease-induced pluripotent stem cells exhibit impaired neurodifferentiation.
Search Criteria
beta N Acetylhexosaminidases