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Multicolor SYNGAP1 Alternative Splice Reporters

Value Proposition· High sensitivity: detection of specific splicing events · High throughput: can assess multiple splicing scenarios due to tri-chromatic and tetra-chromatic reporter plasmids · Improved SYNGAP1 treatment research· Reduced time to assess candidate treatments Technology DescriptionResearchers at Johns Hopkins...

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

Keywords(s):

Category(s): Technology Classifications > Research Tools > Vectors & Plasmids, Clinical and Disease Specializations > Psychiatry > Autism Spectrum Disorder, Clinical and Disease Specializations > Psychiatry > Schizophrenia

SYNGAP antisense oligonucleotide as therapy for human cognitive disorders

Unmet Need: Recent human genetic studies have suggested that mutations in the SYNGAP1 gene are linked to intellectual disability (ID), autism spectrum disorders (ASD), neurodevelopmental disorders, high rates of epilepsy, and schizophrenia. SYNGAP1 is a gene that encodes SynGAP, a GTPase-activating protein that is highly enriched in nerve cells in the...

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

Keywords(s): Biologics, CNS and Neurological Disorders, Cognitive Impairment, Disease Indication, Gene Therapy, Nucleic Acid, Single, Target, Therapeutic Matter, Therapeutic Substance, Therapeutics, Therapy Type

Category(s): Clinical and Disease Specializations > Psychiatry > Autism Spectrum Disorder, Clinical and Disease Specializations > Psychiatry > ADHD, Clinical and Disease Specializations > Psychiatry > Mood Disorders, Technology Classifications > Therapeutic Modalities > Gene Therapies