U1 snRNA interactions with deep intronic sequences regulate splicing of multiple exons of spinal muscular atrophy genes.

Autor: Ottesen EW; Department of Biomedical Sciences, Iowa State University, Ames, IA, United States., Singh NN; Department of Biomedical Sciences, Iowa State University, Ames, IA, United States., Seo J; Department of Biomedical Sciences, Iowa State University, Ames, IA, United States., Singh RN; Department of Biomedical Sciences, Iowa State University, Ames, IA, United States.

Publikováno v: Frontiers in neuroscience [Front Neurosci] 2024 Jul 12; Vol. 18, pp. 1412893. Date of Electronic Publication: 2024 Jul 12 (Print Publication: 2024).

A super minigene with a short promoter and truncated introns recapitulates essential features of transcription and splicing regulation of the SMN1 and SMN2 genes.

Autor: Ottesen EW; Department of Biomedical Sciences, Iowa State University, Ames, IA 50011, USA., Seo J; Department of Biomedical Sciences, Iowa State University, Ames, IA 50011, USA., Luo D; Department of Biomedical Sciences, Iowa State University, Ames, IA 50011, USA., Singh NN; 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] 2024 Apr 24; Vol. 52 (7), pp. 3547-3571.

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.

The First Orally Deliverable Small Molecule for the Treatment of Spinal Muscular Atrophy.

Autor: Singh RN; Department of Biomedical Sciences, Iowa State University, Ames, IA, USA., Ottesen EW; Department of Biomedical Sciences, Iowa State University, Ames, IA, USA., Singh NN; Department of Biomedical Sciences, Iowa State University, Ames, IA, USA.

Publikováno v: Neuroscience insights [Neurosci Insights] 2020 Nov 23; Vol. 15, pp. 2633105520973985. Date of Electronic Publication: 2020 Nov 23 (Print Publication: 2020).

A survey of transcripts generated by spinal muscular atrophy genes.

Autor: Singh NN; Department of Biomedical Science, Iowa State University, Ames, IA, 50011, United States of America., Ottesen EW; Department of Biomedical Science, Iowa State University, Ames, IA, 50011, United States of America., Singh RN; Department of Biomedical Science, Iowa State University, Ames, IA, 50011, United States of America. Electronic address: singhr@iastate.edu.

Publikováno v: Biochimica et biophysica acta. Gene regulatory mechanisms [Biochim Biophys Acta Gene Regul Mech] 2020 Aug; Vol. 1863 (8), pp. 194562. Date of Electronic Publication: 2020 May 06.