Reengineering a transmembrane protein to treat muscular dystrophy using exon skipping

Exon skipping uses antisense oligonucleotides as a treatment for genetic diseases. The antisense oligonucleotides used for exon skipping are designed to bypass premature stop codons in the target RNA and restore reading frame disruption. Exon skipping is currently being tested in humans with dystrop...

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Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:J Clin Invest
Prif Awduron: Gao, Quan Q., Wyatt, Eugene, Goldstein, Jeff A., LoPresti, Peter, Castillo, Lisa M., Gazda, Alec, Petrossian, Natalie, Earley, Judy U., Hadhazy, Michele, Barefield, David Y., Demonbreun, Alexis R., Bönnemann, Carsten, Wolf, Matthew, McNally, Elizabeth M.
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: American Society for Clinical Investigation 2015
Pynciau:
Research Article
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC4639981/
https://ncbi.nlm.nih.gov/pubmed/26457733
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI82768
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