Propionic and Methylmalonic Acidemia: Antisense Therapeutics for Intronic Variations Causing Aberrantly Spliced Messenger RNA

We describe the use of antisense morpholino oligonucleotides (AMOs) to restore normal splicing caused by intronic molecular defects identified in methylmalonic acidemia (MMA) and propionic acidemia (PA). The three new point mutations described in deep intronic regions increase the splicing scores of...

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Main Authors: Rincón, A. , Aguado, C. , Desviat, L. R. , Sánchez-Alcudia, R. , Ugarte, M. , Pérez, B. 
Format: Artigo
Jezik:Inglês
Izdano: American Society of Human Genetics 2007
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Article
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC2276355/
https://ncbi.nlm.nih.gov/pubmed/17966092
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