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Comment & Response
December 2016

Doubts About Therapy for Neurological Diseases With Antisense Oligonucleotides

Author Affiliations
  • 1Downstate Medical Center, State University of New York, Brooklyn

Copyright 2016 American Medical Association. All Rights Reserved. Applicable FARS/DFARS Restrictions Apply to Government Use.

JAMA Neurol. 2016;73(12):1502. doi:10.1001/jamaneurol.2016.4332

To the Editor The review by Corey1 and the accompanying editorial paint a very positive picture about the potential of antisense oligonucleotides (ASOs) to treat neurological diseases such as Friedrich ataxia,2 spinal muscular atrophy,3 and Duchenne muscular dystrophy. Although the principle on which ASO-based treatment is based is promising, a review of the literature, however, reveals critical lacunae in the data that have been used to claim efficacy.