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

Hum Gene Ther Clin Dev. 2014 Sep;25(3):112-33. doi: 10.1089/humc.2014.2513.
No abstract available

Publication types

  • Clinical Trial, Phase I
  • Clinical Trial, Phase II

MeSH terms

  • Cohort Studies
  • DNA, Complementary / genetics
  • DNA, Complementary / metabolism
  • Dependovirus / genetics*
  • Female
  • Follow-Up Studies
  • Gene Transfer Techniques*
  • Genetic Therapy / methods*
  • Genetic Vectors / genetics*
  • Humans
  • Male
  • Serogroup
  • alpha 1-Antitrypsin / genetics*
  • alpha 1-Antitrypsin / metabolism
  • alpha 1-Antitrypsin Deficiency / therapy*

Substances

  • DNA, Complementary
  • alpha 1-Antitrypsin