Researchers make stem cells with disease genes

By Mass High Tech Staff

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Researchers from the Harvard Stem Cell Institute (HSCI) and Children’s Hospital Boston report they have developed a line of stem cells that carry the identical genes for a group of highly heritable diseases.

The lines were made using a new induced pluripotent stem cell (iPS) technique. The process involved taking skin and bone marrow cells from donor patients and reprogramming them to turn into stem cells using a virus to deliver transformative genes into their DNA.

The work was performed by lead investigator George Daley, HSCI researcher and associate director of the Stem Cell Program at Children’s Hospital Boston, and his fellow scientists, Konrad Hochedlinger and Chad Cowan, of the HSCI.

The cells — which contain the exact genetic mutations that lead to the diseases — were taken from patients with Down Syndrome, Parkinson’s disease, Gaucher’s, Type 1 diabetes, Huntington’s Disease, Muscular Dystrophy and Lesch-Nyhan syndrome, which is a debilitating enzyme deficiency disorder.

The cell library will be available for research to any institution and will be stored at HSCI’s facility under development at MGH.

The paper was published in the August 6th online edition of the journal Cell.

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