Antisense Oligonucleotide Therapies for Neurodegenerative Diseases.

ABSTRACT: Antisense oligonucleotides represent a novel therapeutic platform for the discovery of medicines that have the potential to treat most neurodegenerative diseases. Antisense drugs are currently in development for the treatment of amyotrophic lateral sclerosis, Huntington's disease, and Alzheimer's disease, and multiple research programs are underway for additional neurodegenerative diseases. One antisense drug, nusinersen, has been approved for the treatment of spinal muscular atrophy. Importantly, nusinersen improves disease symptoms when administered to symptomatic patients rather than just slowing the progression of the disease. In addition to the benefit to spinal muscular atrophy patients, there are discoveries from nusinersen that can be applied to other neurological diseases, includi

SUBMITTER: Bennett CF 

PROVIDER: S-EPMC7427431 | biostudies-literature | 2019 Jul

REPOSITORIES: biostudies-literature