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SYNGAP1 splice-switching antisense oligonucleotide as therapy for human cognitive disorders

Unmet Need: SYNGAP1-related Intellectual Disability (SRID, MRD5) is a severe neurodevelopmental disorder (NDD) characterized by encephalopathy, intellectual disability (ID), autism spectrum disorder (ASD), and epilepsy and accounts for 0.5-1% of all NDDs and ~1% of the ~200 million ID cases worldwide (SYNGAP Research Fund, 2020). Supportive management...

Published: 3/14/2025 | Inventor(s): Richard Huganir, Yoichi Araki, Ingie Hong, Richard Johnson, Yinuo Han

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Category(s): Technology Classifications > Research Tools > Vectors & Plasmids