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Potential therapeutic applications of antisense morpholino oligonucleotides in modulation of splicing in primary immunodeficiency diseases

Highly complementary antisense morpholino oligonucleotides (AMOs) can bind to pre-mRNA and modulate splicing site selection. This offers a powerful tool to regulate splicing process, such as correcting subtypes of splicing mutations and nonsense mutations and reprogramming alternative splicing proce...

Ausführliche Beschreibung

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Bibliographische Detailangaben
Hauptverfasser: Du, Liutao, Gatti, Richard A.
Format: Artigo
Sprache:Inglês
Veröffentlicht: 2010
Schlagworte:
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3061259/
https://ncbi.nlm.nih.gov/pubmed/21147113
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.jim.2010.12.001
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