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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...
Gorde:
| Argitaratua izan da: | Mol Ther Nucleic Acids |
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| Egile Nagusiak: | , , , , |
| Formatua: | Artigo |
| Hizkuntza: | Inglês |
| Argitaratua: |
Nature Publishing Group
2015
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| Gaiak: | |
| Sarrera elektronikoa: | 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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