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DNA nanoparticle-mediated ABCA4 delivery rescues Stargardt dystrophy in mice
Mutations in the photoreceptor-specific flippase ABCA4 are associated with Stargardt disease and many other forms of retinal degeneration that currently lack curative therapies. Gene replacement is a logical strategy for ABCA4-associated disease, particularly given the current success of traditional...
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| Auteurs principaux: | , , , , |
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| Format: | Artigo |
| Langue: | Inglês |
| Publié: |
American Society for Clinical Investigation
2012
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| Sujets: | |
| Accès en ligne: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3428101/ https://ncbi.nlm.nih.gov/pubmed/22886305 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI64833 |
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