Focus10TH ANNIVERSARY SERIES

Wrangling RNA: Antisense oligonucleotides for neurological disorders

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Science Translational Medicine  25 Sep 2019:
Vol. 11, Issue 511, eaay2069
DOI: 10.1126/scitranslmed.aay2069

Abstract

Effective treatment of spinal muscular atrophy with antisense oligonucleotide therapy opens the door to treating other neurological disorders with this approach.

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