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Abstract

Described herein are methods for treating a subject having or at risk of developing a disorder affecting the central nervous system (CNS) (e.g., a neurocognitive disorder, a neuromuscular disorder, or a neurodegenerative disorder (such as FTD, AD, PD, dementia with Lewy bodies, ALS, or a related neurocognitive or motor neuron disorder) or a lysosomal storage disorder). The methods of the disclosure may include administering an adeno-associated viral (AAV) vector that expresses a therapeutic protein (e.g., whose deficiency or lack of activity is associated with the disorder or whose supplementation is likely to benefit the patient). The AAV vectors of the disclosure may be administered, e.g., in particular quantities and by way of particular routes of administration that achieve gene expression in the CNS while avoiding transduction in peripheral tissues (e.g., liver, lung, and spleen).

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First Claim