Thursday, May 19, 2011

Editing scrambled genes in human stem cells may help realize the promise of stem cell-gene therapy

Source: Salk Institute for Biological Studies
Date: May 19, 2011

Summary:

In principle, genetic engineering is simple, but in practice, replacing a faulty gene with a healthy copy is anything but. Using mutated versions of the lamin A gene as an example to demonstrate the versatility of their virus-based approach, researchers at the Salk Institute for Biological Studies successfully edited a diseased gene in patient-specific induced pluripotent stem cells as well as adult stem cells.

The study, which will be published in the June 3, 2011 issue of Cell Stem Cell but are already available online, demonstrates that the gene-editing approach developed by Salk professor Juan Carlos Izpisúa Belmonte, Ph.D., and his team provides an efficient and safe tool for cell engineering and opens the way for gene editing-based stem cell therapies suitable for clinical applications.