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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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Библиографические подробности
Главные авторы: Eckenfelder, Agathe, Tordo, Julie, Babbs, Arran, Davies, Kay E, Goyenvalle, Aurélie, Danos, Olivier
Формат: Artigo
Язык:Inglês
Опубликовано: Nature Publishing Group 2012
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Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC3390224/
https://ncbi.nlm.nih.gov/pubmed/23344083
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2012.24
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