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CEP290 gene transfer rescues Leber Congenital Amaurosis cellular phenotype
Mutations in CEP290 are the most common cause of Leber congenital amaurosis (LCA), a severe inherited retinal degenerative disease for which there is currently no cure. Autosomal recessive CEP290-associated LCA is a good candidate for gene-replacement therapy, and cells derived from affected individ...
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| Asıl Yazarlar: | , , , , , , , , , , |
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| Materyal Türü: | Artigo |
| Dil: | Inglês |
| Baskı/Yayın Bilgisi: |
2014
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| Konular: | |
| Online Erişim: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4188442/ https://ncbi.nlm.nih.gov/pubmed/24807808 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2014.39 |
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