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Splicing therapeutics in SMN2 and APOB

Splicing therapeutics are defined as the deliberate modification of RNA splicing to achieve therapeutic goals. Various techniques for splicing therapeutics have been described, and most of these involve the use of antisense oligonucleotide-based compounds that target key elements in the pre-mRNA to...

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Detalhes bibliográficos
Main Authors: Khoo, Bernard, Krainer, Adrian R
Formato: Artigo
Idioma:Inglês
Publicado em: 2009
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3140428/
https://ncbi.nlm.nih.gov/pubmed/19330716
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