Search Results - richard+huganir

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SYNGAP1 Mouse Models

Unmet NeedIt is estimated that there are 177 million people in the world affected by non-syndromic intellectual disability and of that group 1% (or 1.77 million people) have a mutation in the SYNGAP1 gene (see SynGAP Research Fund). The SYNGAP1 gene is essential for mental health, and it is known that mutations in the gene lead to severe intellectual...

Published: 5/10/2024 | Inventor(s): Richard Huganir

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Category(s): Technology Classifications > Research Tools > Animal Models

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: 5/9/2024 | 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

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: 5/10/2024 | Inventor(s): Richard Huganir, Yoichi Araki, Ingie Hong, Richard Johnson, Yinuo Han

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

Gene therapy for SYNGAP1 encephalopathy and SYNGAP1-related disorders

Published: 5/10/2024 | Inventor(s): Richard Huganir, Ingie Hong, Yoichi Araki, Richard Johnson

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Category(s): Clinical and Disease Specializations > Rare Diseases, Clinical and Disease Specializations > Neurology, Technology Classifications > Therapeutic Modalities > Gene Therapies

SEP-GluA1 mouse line

Unmet Need:AMPA-type glutamate receptors (AMPARs) are crucial molecules to study to understand the function the nervous system. These receptors mediate the majority of fast excitatory activities in the mammalian brain, and their regulation is regarded as a key mechanism underlying long-lasting changes that give rise to learning and memory. Elucidating...

Published: 5/10/2024 | Inventor(s): Richard Huganir

Keywords(s):

Category(s): Clinical and Disease Specializations > Neurology, Technology Classifications > Research Tools > Animal Models, Technology Classifications > Research Tools

Anti-TARP Gamma 8 pAb

Polyclonal Antibody specific for Transmembrane AMPA Receptor Protein (TARP)JHU REF: [C12231]Invention novelty: This technology is an anti-TARP [transmembrane alpha-amino-3-hydroxyl-5-isoxazolepropionate (AMPA) receptor protein] gamma 8 polyclonal antibody.Value Proposition: In many neurological diseases, AMPA receptor function and structure are altered. ...