Successful Correction of the Human Cooley's Anemia β-Thalassemia Major Phenotype Using a Lentiviral Vector Flanked by the Chicken Hypersensitive Site 4 Chromatin Insulator

• Published in: Annals of the New York Academy of Sciences Vol. 1054; no. 1; pp. 238 - 249
• Main Authors: MALIK, PUNAM, ARUMUGAM, PARITHA I., YEE, JING-KUAN, PUTHENVEETIL, GEETHA
• Format: Journal Article
• Language: English
• Published: Oxford, UK Blackwell Publishing Ltd 01.11.2005
• Subjects: Animals; Apoptosis; beta-Thalassemia - genetics; beta-Thalassemia - therapy; Cell Differentiation; Cells, Cultured - metabolism; Cells, Cultured - transplantation; Chickens - genetics; Colony-Forming Units Assay; Defective Viruses - genetics; DNA Methylation; Erythroid Cells - metabolism; Erythropoiesis; Gene Silencing; Genes, Synthetic; Genetic Therapy; Genetic Vectors - genetics; Globins - genetics; Humans; Insulator Elements - genetics; lentiviral vector; Lentivirus - genetics; Mice; Mice, Inbred NOD; Mice, SCID; Phenotype; Proviruses - genetics; Recombinant Fusion Proteins - biosynthesis; Recombinant Fusion Proteins - genetics; Recombinant Fusion Proteins - physiology; Splenectomy; Terminal Repeat Sequences - genetics; Transduction, Genetic; Transplantation, Heterologous; β-globin; β-thalassemia; β‐thalassemia, lentiviral vector
• ISSN: 0077-8923; 1749-6632
• DOI: 10.1196/annals.1345.030

: β‐Thalassemias are the most common single‐gene disorders and are potentially amenable to gene therapy. While retroviral vectors carrying the human β‐globin cassette were notoriously unstable and expressed poorly, considerable progress has now been made using lentiviral vectors (LVs), which stably...