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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...
| Autores principales: | , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Nature Publishing Group
2015
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4877449/ https://www.ncbi.nlm.nih.gov/pubmed/26325627 http://dx.doi.org/10.1038/mtna.2015.24 |