Consequences of Making the Inactive Active Through Changes in Antisense Oligonucleotide Chemistries

Antisense oligonucleotides are short, single-stranded nucleic acid analogues that can interfere with pre-messenger RNA (pre-mRNA) processing and induce excision of a targeted exon from the mature transcript. When developing a panel of antisense oligonucleotides to skip every dystrophin exon, we foun...

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Detalhes bibliográficos

Principais autores:	Khine Zaw, Kane Greer, May Thandar Aung-Htut, Chalermchai Mitrpant, Rakesh N. Veedu, Sue Fletcher, Steve D. Wilton
Formato:	Artigo
Idioma:	Inglês
Publicado em:	Frontiers Media S.A. 2019-12-01
coleção:	Frontiers in Genetics
Assuntos:	DMD antisense oligonucleotide locked nucleic acid locked nucleic acid/2′-O-methyl mixmer cryptic splice site
Acesso em linha:	https://www.frontiersin.org/article/10.3389/fgene.2019.01249/full
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