Somatic Therapy of a Mouse SMA Model with a U7 snRNA Gene Correcting SMN2 Splicing

Spinal Muscular Atrophy is due to the loss of SMN1 gene function. The duplicate gene SMN2 produces some, but not enough, SMN protein because most transcripts lack exon 7. Thus, promoting the inclusion of this exon is a therapeutic option. We show that a somatic gene therapy using the gene for a modi...

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Detalles Bibliográficos
Autores principales: Odermatt, Philipp, Trüb, Judith, Furrer, Lavinia, Fricker, Roger, Marti, Andreas, Schümperli, Daniel
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2016
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5112044/
https://www.ncbi.nlm.nih.gov/pubmed/27456062
http://dx.doi.org/10.1038/mt.2016.152

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5112044/
https://www.ncbi.nlm.nih.gov/pubmed/27456062
http://dx.doi.org/10.1038/mt.2016.152

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