RNA-based drug discovery for spinal muscular atrophy: a story of small molecules and antisense oligonucleotides

Torroba, B; Macabuag, N; Haisma, EM; O'Neill, A; Herva, ME; Redis, RS; Templin, MV; Black, LE; Fischer, DF

Fischer, DF (通讯作者)，Charles River Labs, Saffron Walden CB10 1XL, Essex, England.

EXPERT OPINION ON DRUG DISCOVERY, 2023; 18 (2): 181

Abstract

IntroductionSpinal Muscular Atrophy (SMA), the second most prevalent autosomal genetic disease affecting infants, is caused by the lack of SMN1, which......

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