AAV Gene Therapy Strategies for Lysosomal Storage Disorders with Central Nervous System Involvement

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Pompe disease gene therapy

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AAV-Mediated Gene Delivery in Adult GM1-Gangliosidosis Mice Corrects Lysosomal Storage in CNS and Improves Survival

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Gene transfer of human acid sphingomyelinase corrects neuropathology and motor deficits in a mouse model of Niemann-Pick type A disease

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Therapeutic Response in Feline Sandhoff Disease Despite Immunity to Intracranial Gene Therapy

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Adeno-associated virus type 4 (AAV4) targets ependyma and astrocytes in the subventricular zone and RMS

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Intravitreal Gene Therapy Reduces Lysosomal Storage in Specific Areas of the CNS in Mucopolysaccharidosis VII Mice

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Complete Correction of Enzymatic Deficiency and Neurochemistry in the GM1-gangliosidosis Mouse Brain by Neonatal Adeno-associated Virus–mediated Gene Delivery

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Gene Transfer Corrects Acute GM2 Gangliosidosis—Potential Therapeutic Contribution of Perivascular Enzyme Flow

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Intracerebroventricular infusion of acid sphingomyelinase corrects CNS manifestations in a mouse model of Niemann–Pick A disease

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Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I

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Combination brain and systemic injections of AAV provide maximal functional and survival benefits in the Niemann-Pick mouse

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