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...

Volledige beschrijving

Bewaard in:
Bibliografische gegevens
Gepubliceerd in:J Clin Invest
Hoofdauteurs: 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.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: American Society for Clinical Investigation 2015
Onderwerpen:
Research Article
Online toegang: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
Tags: Voeg label toe
Geen labels, Wees de eerste die dit record labelt!

Gelijkaardige items