The Cellular Processing Capacity Limits the Amounts of Chimeric U7 snRNA Available for Antisense Delivery

Many genetic diseases are induced by mutations disturbing the maturation of pre-mRNAs, often affecting splicing. Antisense oligoribonucleotides (AONs) have been used to modulate splicing thereby circumventing the deleterious effects of mutations. Stable delivery of antisense sequences is achieved by...

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Detalles Bibliográficos
Autores principales: Eckenfelder, Agathe, Tordo, Julie, Babbs, Arran, Davies, Kay E, Goyenvalle, Aurélie, Danos, Olivier
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2012
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3390224/
https://www.ncbi.nlm.nih.gov/pubmed/23344083
http://dx.doi.org/10.1038/mtna.2012.24

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3390224/
https://www.ncbi.nlm.nih.gov/pubmed/23344083
http://dx.doi.org/10.1038/mtna.2012.24

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