METHODS AND MATERIALS FOR TREATING SYNGAP1-ASSOCIATED NEURODEVELOPMENTAL DISORDERS

Abstract: this document provides methods and materials for treating syngap1-associated neurodevelopmental disorders (ndds; e.g., syngap1-related intellectual disability (srid)). for example, viral vectors (e.g., adeno-associated viral (aav) vectors) that include (e.g., are designed to include) nucleic acid encoding a truncated syngap1 polypeptide (e.g., syngap1-b:�1 polypeptide) are provided. in some cases, one or more viral vectors provided herein (e.g., aav vectors that include nucleic acid encoding a truncated syngap1 polypeptide such as a syngap1-b:�1 polypeptide) can be administered to a mammal (e.g., a human) having, or at risk for developing, a syngap1-associated ndd (e.g., srid) to treat the mammal.

Inventor(s): Ingie Hong, Richard Huganir, Yoichi Araki, Richard C. Johnson

CPC Classification: C12N15/86 (MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media ))

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