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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
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
Keywords(s):
Category(s):
Technology Classifications > Research Tools > Vectors & Plasmids
Gene therapy for SYNGAP1 encephalopathy and SYNGAP1-related 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
,
Ingie Hong
,
Yoichi Araki
,
Richard Johnson
Keywords(s):
Category(s):
Clinical and Disease Specializations > Rare Diseases
,
Clinical and Disease Specializations > Neurology
,
Technology Classifications > Therapeutic Modalities > Gene Therapies
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