Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing α <sub>1</sub> -Antitrypsin: Interim Results

R. Flotte, Terence; C. Trapnell, Bruce; Humphries, Margaret; Carey, Brenna; Calcedo, Roberto; N. Rouhani, Farshid; Campbell‐Thompson, Martha; T. Yachnis, Anthony; A. Sandhaus, Robert; G. McElvaney, Noel; Mueller, Christian; M. Messina, Louis; M. Wilson, James; Brantly, Mark; R. Knop, David; Ye, Guo-jie; D. Chulay, Jeffrey

doi:10.1089/hum.2011.053

Public

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

Published in Human Gene Therapy • May 24, 2011

Authors:

Terence R. Flotte

Bruce C. Trapnell

Margaret Humphries

Abstract

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, thi...

Subject

Adeno-associated virus

Genetic enhancement

Vector (molecular biology)

Research Assistant

AI chat, annotations, notes & similar papers

Finding related papers...

Discussions

(0)

No comments yet

Be the first to share your thoughts!