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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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Autors principals: Khoo, Bernard, Krainer, Adrian R
Format: Artigo
Idioma:Inglês
Publicat: 2009
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3140428/
https://ncbi.nlm.nih.gov/pubmed/19330716
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