
Increasing survival of motor neuron 2, centromeric (SMN2) exon 7 inclusion to express more full-length SMN protein in motor neurons is a promising approach to treat spinal muscular atrophy …
Antisense correction of SMN2 splicing in the CNS rescues …
Jul 12, 2010 · Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model Supplemental Material Files in this Data Supplement: Supp Figures.pdf …
feedback loop regulates splicing of the spinal muscular atrophy ...
Sep 30, 2010 · We now show that SMN protein abundance affects the splicing of SMN2 exon 7, revealing a feedback loop inSMN expression. The reduced SMN protein concentration …
Determinants of Exon 7 Splicing in the Spinal Muscular Atrophy Genes ...
Jan 1, 2006 · Our findings explain the basis of defective SMN2 splicing, illustrate the fine balance between positive and negative determinants of exon identity and alternative splicing, and …
Hua Y, Sahashi K, Hung G, et al. Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model. Genes Dev. 2010: 24(15):1634‐44.
Transcriptional and Splicing Regulation of Spinal Muscular Atrophy Genes
Jan 1, 2017 · With the significance to the development- and tissue-specific splicing regulation of SMN2 exons, a recent report has shown a splicing switch from SMN2 exon 7 skipping to …
splicing silencer in SMN2 intron 6 is critical in spinal muscular ...
Oct 18, 2022 · The combinatory silencing activities of these splicing repressors result in a strong intronic splicing silencer in SMN2 exon 7 splicing. Multiple-antisense oligonucleotide (ASO) …
Antisense correction of SMN2 splicing in the CNS rescues …
Jul 12, 2010 · A comment on this article appears in "Antisense oligonucleotides and spinal muscular atrophy: skipping along." Genes Dev. 2010 Aug 1;24 (15):1574-9.
Molecular Therapeutic Strategies for Spinal Muscular Atrophies…
Jan 1, 2014 · The human genome harbors an additional SMN2 gene (or genes) that produces low levels of full-length SMN but cannot adequately compensate for the loss of SMN1 due to an …
Genes Dev. published online July 12, 2010 € Yimin Hua, Kentaro Sahashi, Gene Hung, et al. € in a type III SMA mouse model Antisense correction of SMN2 splicing in the CNS rescues necrosis