Diverse targets of SMN2-directed splicing-modulating small molecule therapeutics for spinal muscular atrophy.

Autor: Ottesen EW; Department of Biomedical Sciences, Iowa State University, Ames, IA 50011, USA., Singh NN; Department of Biomedical Sciences, Iowa State University, Ames, IA 50011, USA., Luo D; Department of Biomedical Sciences, Iowa State University, Ames, IA 50011, USA., Kaas B; Department of Biomedical Sciences, Iowa State University, Ames, IA 50011, USA., Gillette BJ; Department of Biomedical Sciences, Iowa State University, Ames, IA 50011, USA., Seo J; Department of Biomedical Sciences, Iowa State University, Ames, IA 50011, USA., Jorgensen HJ; Department of Biomedical Sciences, Iowa State University, Ames, IA 50011, USA., Singh RN; Department of Biomedical Sciences, Iowa State University, Ames, IA 50011, USA.

Publikováno v: Nucleic acids research [Nucleic Acids Res] 2023 Jul 07; Vol. 51 (12), pp. 5948-5980.