RNA-targeted Therapeutics for ALS

Neurotherapeutics : the Journal of the American Society for Experimental NeuroTherapeutics
Linga V Reddy, Timothy M Miller

Abstract

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease leading to cell death of predominantly motor neurons. Despite extensive research in this disease, finding a way to slow the progress of the disease has been challenging. RNA-targeted therapeutic approaches, including small interfering RNA and antisense oligonucleotides are being developed for genetic forms of ALS. ALS provides an unique opportunity for the use of RNA inhibition strategies given a well-defined animal model, extensive available information regarding the causative genes, and recent experience in phase 1 clinical trial.

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Citations

Mar 22, 2015·Neurotherapeutics : the Journal of the American Society for Experimental NeuroTherapeutics·Stacey A Sakowski, Eva L Feldman
Oct 22, 2016·European Journal of Medicinal Chemistry·Vijay KumarMd Imtaiyaz Hassan
Feb 17, 2017·Biochemistry·Mia K MihailovicLydia M Contreras
Jan 19, 2018·Biomedicines·Stéphane Mathis, Gwendal Le Masson
Jan 29, 2017·Cold Spring Harbor Perspectives in Medicine·Tania F Gendron, Leonard Petrucelli

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