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Updated: Mar 9, 2026

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The Antisense Transcript SMN-AS1 Regulates SMN Expression and Is a Novel Therapeutic Target for Spinal Muscular

Constantin d'Ydewalle1, Daniel M Ramos2, Noah J Pyles1

  • 1Department of Neurology, Johns Hopkins University School of Medicine, 855 North Wolfe Street, Baltimore, MD 21205, USA.

Neuron
|December 27, 2016
PubMed

Keywords:
SMA therapeuticsSMNantisense oligonucleotideslong non-coding RNAspinal muscular atrophy

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