Tag Archives: Stem Cells

Breakthrough in Stem Cell Research Provides Means for a Novel Form of Gene Therapy

Recently, scientists have discovered a technique to “reprogram” adult human and mouse cells to revert into induced Pluripotent Stem (iPS) cells. These cells exhibit the defining characteristics of ES cells and are essentially indistinguishable by all standard tests used to verify developmental stage in cells. The technique employs a retrovirus to insert four inappropriately active genes into the adult genome, initiating massive nuclear reprogramming into an ES cell-like state. Former techniques, such as nuclear transfer employed to create Dolly, ubiquitously failed because the adult DNA (chromatin) structure was not reprogrammed to resemble the structure of embryonic chromatin, resulting in improper gene expression.

This innovative and new technique must undergo significant testing before it can be used in any form of clinical trial: in particular, the use of a retrovirus causes a high risk of cancer, since the virus integrates randomly into the genome. However, this technique provides a novel vector for gene therapy which has the potential to be developed into treatments not limited to: cancer treatment, replacement therapy in degenerative diseases and organ replacement without donor-rejection complications. In mice, Rudolf Jaensich of MIT has already harnessed this technology to physiologically cure sickle-celled mice free of any initially discernible side effects.

Posted by Milly Ray, PhD candidate, Harvard Medical School