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An AAV Dual Vector Strategy Ameliorates the Stargardt Phenotype in Adult Abca4(−/−) Mice
The recent approval in the United States of the first adeno-associated viral (AAV) vector for the treatment of an inherited retinal degeneration validates this approach for the treatment of many other diseases. A major limiting factor continues to be the size restriction of the AAV transgene at unde...
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| Pubblicato in: | Hum Gene Ther |
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| Autori principali: | , , , , , , |
| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
Mary Ann Liebert, Inc., publishers
2019
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| Soggetti: | |
| Accesso online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6909730/ https://ncbi.nlm.nih.gov/pubmed/30381971 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2018.156 |
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