Article
Successful correction of the human Cooley's anemia beta-thalassemia major phenotype using a lentiviral vector flanked by the chicken hypersensitive site 4 chromatin insulator.
Annals of the New York Academy of Sciences - 1 Jan 2005
Malik Punam, Arumugam Paritha I, Yee Jing-Kuan, Puthenveetil Geetha
Abstract excerpt
beta-Thalassemias are the most common single-gene disorders and are potentially amenable to gene therapy. While retroviral vectors carrying the human beta-globin cassette were notoriously unstable and expressed poorly, considerable progress has now been made using lentiviral vectors (LVs), which stably transmit the beta-globin expression cassette. Mouse studies using LVs have shown correction of the...
Topics
- Animals
- Apoptosis
- Cell Differentiation
- Cells, Cultured
- Chickens
- Colony-Forming Units Assay
- DNA Methylation
- Defective Viruses
- Erythroid Cells
- Erythropoiesis