Gene therapy for stxbp1 encephalopathy
AssigneeBaylor College of Medicine
InventorsMingshan Xue, Wu Chen
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Abstract
The disclosure concerns compositions and methods for treatment of encephalopathies, including encephalopathies caused by, or associated with, and STXBP1 haploinsufficiency or mutation. Compositions and methods provided herein encompass AAV particles that induce expression of STXBP1, including inducing the expression of STXBP1 in central and/or peripheral nervous system cells.
Claims35
An adeno-associated virus (AAV) particle comprising a nucleic acid comprising a sequence encoding an STXBP1 gene product.
An AAV particle comprising a nucleic acid encoding an STXBP1 gene product having at least 90% sequence identity with SEQ ID NO:2.
A method of treating an individual with an STXBP1 encephalopathy comprising administering a therapeutically effective amount of an AAV particle comprising a nucleic acid encoding an STXBP1 gene product having at least 95% sequence identity with SEQ ID NO:2.
Dependencies are inferred from the claim text.