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Adeno-Associated Virus Gene Therapy in a Sheep Model of Tay-Sachs Disease.
Mucopolysaccharidosis-like phenotype in feline Sandhoff disease and partial correction after AAV gene therapy.
A Safe and Reliable Technique for CNS Delivery of AAV Vectors in the Cisterna Magna.
Intravenous delivery of adeno-associated viral gene therapy in feline GM1 gangliosidosis.
Adeno-Associated Virus Delivery Limitations for Neurological Indications.