Doubts About Therapy for Neurological Diseases With Antisense Oligonucleotides

Satyakam Bhagavati, MD, JAMA Neurol. Published online October 31, 2016. doi:10.1001/jamaneurol.2016.4332

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.

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 Doubts About Therapy for Neurological Diseases With Antisense Oligonucleotides


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