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Glucocerebrosidase 1 (GBA1) Knockout SH-SY5Y Cell Line
JHU Ref #: C18058 Value Proposition:GBA1 knockout SH-SY5Y human neuroblastoma cell lineGenerated using CRISPR/Cas9 KO technologyUnavailable from any other sourceNo observable off-target effectsUnmet Need:It has been demonstrated that GBA1 deficiency contributes to the aggregation of α-synuclein (α-syn) and the transition from monomeric α-syn to α-syn...
Published: 3/14/2025
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Inventor(s):
Han Seok Ko
Keywords(s):
Category(s):
Inhibition of rip kinases for treating neurodegenerative disorders
Unmet NeedNeurodegenerative disorders are a heterogeneous group that includes Alzheimer’s disease (AD), Parkinson’s disease (PD), amyotrophic lateral sclerosis (ALS), multiple sclerosis (MS), dementia, and Huntington’s disease. These disorders result from progressive degeneration of the structure, function, and network of neurons in the central nervous...
Published: 3/13/2025
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Inventor(s):
Seulki Lee
,
Han Seok Ko
,
Ted Dawson
,
Martin Pomper
,
Donghoon Kim
,
Yumin Oh
,
Seung-Hwan Kwon
,
Yong Joo Park
Keywords(s):
Alzheimer's Disease
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Antagonists/Inhibitors
,
CNS and Neurological Disorders
,
Disease Indication
,
Neurodegenerative Diseases
,
Parkinson's Disease
,
Small Molecules
,
Therapeutic Matter
,
Therapeutic Substance
,
Therapeutics
Category(s):
Technology Classifications > Therapeutic Modalities > Small Molecules
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Clinical and Disease Specializations > Neurology > Neurodegeneration
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Clinical and Disease Specializations > Neurology > Alzheimer's Disease
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Clinical and Disease Specializations > Neurology > Parkinson’s Disease
Platform for drug screening in human neurons
Unmet NeedThe WHO has predicted that neurological disorders (NDs) will become the second leading cause of death in 20 years after cardiovascular diseases. Various proteins have been identified to aggregate and accumulate in the brain depending on the indication: amyloid-beta or tau in Alzheimer’s Disease, alpha-synuclein in Parkinson’s disease, and...
Published: 3/14/2025
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Inventor(s):
Gabsang Lee
,
Minseong Kim
,
Eun Ra
,
Han Seok Ko
,
Xiaobo Mao
Keywords(s):
Category(s):
Clinical and Disease Specializations > Neurology > Parkinson’s Disease
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Technology Classifications > Research Tools > Cell Lines
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Technology Classifications > Therapeutic Modalities
Identification of LAG3 as the α-synuclein Neuronal Transmission Receptor
Unmet Need: Parkinson’s disease (PD) is the second most common neurodegenerative disorder and leads to slowness of movement, tremor, rigidity, and, in the later stages of PD, cognitive impairment. Pathologically, PD is characterized by the accumulation of α-synuclein in Lewy bodies and neurites. Emerging evidence indicates that the pathogenesis of Parkinson's...
Published: 3/13/2025
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Inventor(s):
Ted Dawson
,
Valina Dawson
,
Han Seok Ko
,
Xiaobo Mao
,
Dario Vignali
,
Creg Workman
Keywords(s):
Biomarker
,
CNS and Neurological Disorders
,
Disease Indication
,
Mechanism-of-action Biomarker
,
Parkinson's Disease
,
Therapeutic Matter
,
Therapeutics
Category(s):
Clinical and Disease Specializations
,
Clinical and Disease Specializations > Neurology > Parkinson's Disease
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Technology Classifications > Diagnostics > Biomarkers
,
Technology Classifications > Therapeutic Modalities > Targets
,
Technology Classifications > Diagnostics
,
Clinical and Disease Specializations > Neurology
,
Technology Classifications > Therapeutic Modalities
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