Intravitreal Injection of Splice-switching Oligonucleotides to Manipulate Splicing in Retinal Cells

Leber congenital amaurosis is a severe hereditary retinal dystrophy responsible for neonatal blindness. The most common disease-causing mutation (c.2991+1655A>G; 10–15%) creates a strong splice donor site that leads to insertion of a cryptic exon encoding a premature stop codon. Recently, we repo...

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Bibliografiska uppgifter
I publikationen:Mol Ther Nucleic Acids
Huvudupphovsmän: Gérard, Xavier, Perrault, Isabelle, Munnich, Arnold, Kaplan, Josseline, Rozet, Jean-Michel
Materialtyp: Artigo
Språk:Inglês
Publicerad: Nature Publishing Group 2015
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Original Article
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC4877449/
https://ncbi.nlm.nih.gov/pubmed/26325627
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2015.24
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