|
Antisense Oligonucleotides to Prevent Genetic Mis-Splicing for ALS/FTD Therapeutics
SHORT DESCRIPTION
Antisense oligonucleotides (ASOs) that correct TDP‑43–dependent mis‑splicing of KCNQ2 thereby reducing neuronal hyperexcitability in ALS/FTD and related TDP‑43 proteinopathies.
INVENTORS
Evangelos Kiskinis*
Northwestern University Feinberg School of Medicine, Department of Neurology
Jonathan...
Published: 5/12/2026
|
Updated: 5/7/2026
|
Inventor(s):
Keywords(s): ALS - Amyotrophic Lateral Sclerosis, FTD - Frontotemporal Dementia, Neurodegenerative disease, Neurologic disease, Neurology, Nucleic Acids, Rare diseases, Targeted therapy, Therapeutics
Category(s): Life Sciences > Therapeutics
|
|
Therapeutic Intervention for ALS and Dementia
NU 2021-058
INVENTORS
Evangelos Kiskinis*
Juan Alberto Ortega Cano Marco Boccitto
SHORT DESCRIPTION
Design of an RNA-based oligonucleotide that protects from dipeptide repeat protein toxicity for the treatment of ALS and FTD.
BACKGROUND
Amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) are devastating neurodegenerative diseases...
Published: 5/7/2026
|
Updated: 1/22/2025
|
Inventor(s):
Keywords(s): ALS - Amyotrophic Lateral Sclerosis, CNS - Central Nervous System, DPR - Dipeptide Repeat Proteins, FTD - Frontotemporal Dementia, Neurodegenerative disease, Neurology, RNA - Ribonucleic Acid, Targeted therapy, Therapeutics
Category(s): Life Sciences > Therapeutics
|
|
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: 4/13/2026
|
Updated: 10/17/2024
|
Inventor(s):
Keywords(s): ALS - Amyotrophic Lateral Sclerosis, FTD - Frontotemporal Dementia, Neurodegenerative disease, Neurology, Nucleic Acids, Rare diseases, Targeted therapy, Therapeutics
Category(s): Life Sciences > Therapeutics
|