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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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Detalhes bibliográficos
Publicado no:Hum Gene Ther
Main Authors: McClements, Michelle E., Barnard, Alun R., Singh, Mandeep S., Charbel Issa, Peter, Jiang, Zhichun, Radu, Roxana A., MacLaren, Robert E.
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc., publishers 2019
Assuntos:
Acesso em linha: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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