BioCentury
ARTICLE | Tools & Techniques

Putting pluripotent pieces together

November 3, 2011 7:00 AM UTC

An international team has developed a method for genetically altering induced pluripotent stem cells that could be useful for treating inherited diseases.1 The team now plans to scale up the method and will need to show the cells are safe for long-term use.

A potential roadmap for using induced pluripotent stem (iPS) cells as therapeutics for genetic diseases has been in place for at least five years2 and involves first harvesting accessible cells such as fibroblasts from the patient and converting them into iPS cells. Next, the patient's genetic defect is corrected in those iPS cells. Finally, the corrected iPS cells are differentiated into the desired cell type and transplanted back into the patient, in which the cells engraft and proliferate in the local tissue to treat the disease...