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A Preclinical Study in Rhesus Macaques for Cystic Fibrosis to Assess Gene Transfer and Transduction by AAV1 and AAV5 with a Dual-Luciferase Reporter System

Cystic fibrosis (CF) is an autosomal recessive disease that is potentially treatable by gene therapy. Since the identification of the gene encoding CF transmembrane conductance regulator, a number of preclinical and clinical trials have been conducted using the first generation of adeno-associated v...

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Publicado en:Hum Gene Ther Clin Dev
Autores principales: Guggino, William B., Benson, Janet, Seagrave, JeanClare, Yan, Ziying, Engelhardt, John, Gao, Guangping, Conlon, Thomas J., Cebotaru, Liudmila
Formato: Artigo
Lenguaje:Inglês
Publicado: Mary Ann Liebert, Inc. 2017
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Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC5655841/
https://ncbi.nlm.nih.gov/pubmed/28726496
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/humc.2017.067
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