Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing α(1)-Antitrypsin: Interim Results

Recombinant adeno-associated virus (rAAV) vectors offer promise for the gene therapy of α(1)-antitrypsin (AAT) deficiency. In our prior trial, an rAAV vector expressing human AAT (rAAV1-CB-hAAT) provided sustained, vector-derived AAT expression for >1 year. In the current phase 2 clinical trial,...

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Библиографические подробности
Главные авторы: Flotte, Terence R., Trapnell, Bruce C., Humphries, Margaret, Carey, Brenna, Calcedo, Roberto, Rouhani, Farshid, Campbell-Thompson, Martha, Yachnis, Anthony T., Sandhaus, Robert A., McElvaney, Noel G., Mueller, Christian, Messina, Louis M., Wilson, James M., Brantly, Mark, Knop, David R., Ye, Guo-jie, Chulay, Jeffrey D.
Формат: Artigo
Язык:Inglês
Опубликовано: Mary Ann Liebert, Inc. 2011
Предметы:
Brief Reports
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC3205788/
https://ncbi.nlm.nih.gov/pubmed/21609134
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2011.053
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