Homologous recombination in human iPS and ES cells for use in gene correction therapy

• Published in: Drug discovery today Vol. 15; no. 5; pp. 198 - 202
• Main Author: Nakayama, Manabu
• Format: Journal Article
• Language: English
• Published: Kidlington Elsevier Ltd 01.03.2010; Elsevier
• Subjects: Animals; Biological and medical sciences; Cell Culture Techniques - methods; Cell Culture Techniques - trends; Embryonic Stem Cells - cytology; Embryonic Stem Cells - physiology; General pharmacology; Genetic Therapy - methods; Genetic Therapy - trends; Humans; Induced Pluripotent Stem Cells - cytology; Induced Pluripotent Stem Cells - physiology; Medical sciences; Pharmaceutical technology. Pharmaceutical industry; Pharmacology. Drug treatments; Recombination, Genetic; Targeted Gene Repair - methods; Targeted Gene Repair - trends; Human; Homology; Pharmaceutical technology; Gene therapy; Induced plutipotent stem cell
• ISSN: 1359-6446; 1878-5832
• DOI: 10.1016/j.drudis.2010.01.006

The emergence of induced pluripotent stem (iPS) cell technology has shifted gene correction therapy toward reality. Crucial issues are ensuring the safety of using iPS cell technology in patients and discovering how best to transfer genetically manipulated iPS cells back into patients. One key issue that has hindered progress of gene correction therapy, however, is the inability to achieve efficient homologous recombination in human iPS cells. This review focuses on recently developed technologies that aim to improve homologous recombination in human embryonic stem cells and on their application to iPS cells.