Article
Sustained expression of alpha1-antitrypsin after transplantation of manipulated hematopoietic stem cells.
American journal of respiratory cell and molecular biology - 1 Aug 2008
Wilson Andrew A, Kwok Letty W, Hovav Avi-Hai, Ohle Sarah J, Little Frederic F, Fine Alan, Kotton Darrell N
Abstract excerpt
Inherited mutations in the human alpha(1)-antitrypsin (AAT) gene lead to deficient circulating levels of AAT protein and a predisposition to developing emphysema. Gene therapy for individuals deficient in AAT is an attractive goal, because transfer of a normal AAT gene into any cell type able to secrete AAT should reverse deficient AAT levels and attenuate progression of lung disease. Here we present an approach...
Topics
- Animals
- Cell Line
- Genes, Reporter
- Genetic Vectors
- Hematopoietic Stem Cell Transplantation
- Hematopoietic Stem Cells
- Humans
- Lentivirus
- Lung
- Mice
- Multipotent Stem Cells
- Mutation
- Transduction, Genetic