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 |