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

Maria J. Chiuchiolo, Stephen M. Kaminsky, Dolan Sondhi, Denesy Mancenido, Charleen Hollmann, Ronald Crystal

Research output: Contribution to journalArticle

11 Citations (Scopus)
Original languageEnglish
Pages (from-to)112-133
Number of pages22
JournalHuman Gene Therapy Clinical Development
Issue number3
Publication statusPublished - 1 Jan 2014
Externally publishedYes

ASJC Scopus subject areas

  • Genetics(clinical)

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