Targeting nuclear RNA for in vivo correction of myotonic dystrophy

Antisense oligonucleotides (ASOs) hold promise for gene-specific knockdown in diseases that involve RNA or protein gain-of-function. In the hereditary degenerative disease myotonic dystrophy type 1 (DM1), transcripts from the mutant allele contain an expanded CUG repeat(1–3) and are retained in the...

Descripción completa

Detalles Bibliográficos
Autores principales: Wheeler, Thurman M., Leger, Andrew J., Pandey, Sanjay K., MacLeod, A. Robert, Nakamori, Masayuki, Cheng, Seng H., Wentworth, Bruce M., Bennett, C. Frank, Thornton, Charles A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2012
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4221572/
https://www.ncbi.nlm.nih.gov/pubmed/22859208
http://dx.doi.org/10.1038/nature11362

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4221572/
https://www.ncbi.nlm.nih.gov/pubmed/22859208
http://dx.doi.org/10.1038/nature11362

Ejemplares similares