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Development of rAAV2-CFTR: History of the First rAAV Vector Product to be Used in Humans

The first human gene therapy trials using recombinant adeno-associated virus (rAAV) vectors were performed in cystic fibrosis (CF) patients. Over 100 CF patients were enrolled in 5 separate trials of rAAV2-CFTR administration via nasal, endobronchial, maxillary sinus, and aerosol delivery. Recombina...

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Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:Hum Gene Ther Methods
Prif Awduron: Loring, Heather S., ElMallah, Mai K., Flotte, Terence R.
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: Mary Ann Liebert, Inc. 2016
Pynciau:
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC4834522/
https://ncbi.nlm.nih.gov/pubmed/26895204
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hgtb.2015.150
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