Phase I/II study of intrapleural administration of a serotype rh.10 replication-deficient adeno-associated virus gene transfer vector expressing the human α1-antitrypsin cDNA to individuals with α1-antitrypsin deficiency.

Publication Type Academic Article
Authors Chiuchiolo M, Kaminsky S, Sondhi D, Mancenido D, Hollmann C, Crystal R
Journal Hum Gene Ther Clin Dev
Volume 25
Issue 3
Pagination 112-33
Date Published 09/01/2014
ISSN 2324-8645
Keywords Dependovirus, Gene Transfer Techniques, Genetic Therapy, Genetic Vectors, alpha 1-Antitrypsin, alpha 1-Antitrypsin Deficiency
DOI 10.1089/humc.2014.2513
PubMed ID 25238276
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