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S/MAR-containing DNA nanoparticles promote persistent RPE gene expression and improvement in RPE65-associated LCA
Mutations in genes in the retinal pigment epithelium (RPE) cause or contribute to debilitating ocular diseases, including Leber's congenital amaurosis (LCA). Genetic therapies, particularly adeno-associated viruses (AAVs), are a popular choice for monogenic diseases; however, the limited payloa...
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| Autori principali: | , , , , |
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| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
Oxford University Press
2013
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| Soggetti: | |
| Accesso online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3605833/ https://ncbi.nlm.nih.gov/pubmed/23335596 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddt013 |
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