Enhancement of SMN2 Exon 7 Inclusion by Antisense Oligonucleotides Targeting the Exon

Several strategies have been pursued to increase the extent of exon 7 inclusion during splicing of SMN2 (survival of motor neuron 2) transcripts, for eventual therapeutic use in spinal muscular atrophy (SMA), a genetic neuromuscular disease. Antisense oligonucleotides (ASOs) that target an exon or i...

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Detalles Bibliográficos
Autores principales: Hua, Yimin, Vickers, Timothy A, Baker, Brenda F, Bennett, C. Frank, Krainer, Adrian R
Formato: Texto
Lenguaje:English
Publicado: Public Library of Science 2007
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1820610/
https://www.ncbi.nlm.nih.gov/pubmed/17355180
http://dx.doi.org/10.1371/journal.pbio.0050073

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1820610/
https://www.ncbi.nlm.nih.gov/pubmed/17355180
http://dx.doi.org/10.1371/journal.pbio.0050073

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