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ASOs targeted towards mis-spliced UNC13A
NU2022-200
INVENTORS
Wanhao Chi
Jonathan Watts
Evangelos Kiskinis*
BACKGROUND
Amyotrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative that leads to 1 in 500 deaths in adults. There is no cure to stop or reverse ALS, thus there is a need to better understand its pathobiology and develop effective therapeutics. Northwestern...
Published: 3/30/2026
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Updated: 10/17/2024
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Inventor(s):
Keywords(s): ALS - Amyotrophic Lateral Sclerosis, FTD - Frontotemporal Dementia, Neurodegenerative disease, Neurology, Nucleic Acids, Targeted therapy, Therapeutics
Category(s): Life Sciences > Therapeutics
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