Editors' ChoiceGene Therapy

Interfering in Charcot-Marie-Tooth disease 2D

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Science Translational Medicine  09 Oct 2019:
Vol. 11, Issue 513, eaaz3712
DOI: 10.1126/scitranslmed.aaz3712

Abstract

Patient-tailored RNA interference prevents the onset of Charcot-Marie-Tooth 2D disease.

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