New method of gene repair developed for gene therapy and stem cell therapy combination treatments

Photo credit: HDBuzz

Researchers have found a new way to snip and edit defective genes using pluripotent stem cells and a DNA-slicing protein from a bacteria that causes meningitis. The study, published earlier this week in the Proceedings of the National Academy of Sciences, was a collaboration between researchers from UC Santa Barbara, The University of Wisconsin-Madison and Northwestern University.

A protein called Cas9, found abundantly in the bacteria Neisseria meningiditis, is a precise DNA cutter, much like a sharp pair of scissors. In combination with small RNA molecules, the research team could manipulate the Cas9 protein to cut, remove and replace problem genes in human pluripotent stem cells.

Pluripotent stem cells can regenerate their populations indefinitely and can develop into any kind of human tissue cell, so this approach holds great promise for treating a variety of diseases.

James A Thompson, the study’s principal investigator and a senior faculty member at both UCSB and the University of Wisconsin–Madison said of the study

“This collaboration has taken us further toward realizing the full potential of these cells because we can now manipulate their genomes in a precise, efficient manner.”

This combination of gene therapy and stem cell therapy isn’t new. CIRM has eight teams working on getting gene therapy and stem cell combination treatments into the clinic. A trial for HIV treatment started last month. One for Beta Thalasemia will begin later this year (insert link: ), and one for sickle cell disease will begin early next year.

The more options stem cell scientists have available, the better — because different techniques are likely to work best in certain niches and for certain diseases. This new method provides one more tool in regenerative medicine’s toolbox.

2 thoughts on “New method of gene repair developed for gene therapy and stem cell therapy combination treatments

  1. Blood of our is traveling in every parts of body. Grip of our body parts is strong for cells so, cells of body parts can not mix in blood. So, If we will make cells of any body parts and inject in our body by blood then cells will attach with their related parts. But our body parts are programed by DNA for limited size. so by this we can repair every our body parts.

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