Fanconi anemia - Source: RadsWiki

(Best Syndication News) Amazing research from the Salk Institute has demonstrated that human genetic diseases can be cured using a combination of gene therapy and stem cells. In the past this research has been conducted on mice, but now Salk colleagues and researchers around the world have cured diseased human cells.

Doctor Juan-Carlos Izpisúa Belmonte, Ph.D., professor in the Expression Laboratory and director of the Center of Regenerative Medicine in Barcelona (CMRB), Spain says they have used induced pluripotent stem (iPS) cells, not embryonic stem cells, to cure Fanconi anemia (FA) in human cells. The doctor used the word “cure” to describe their findings.

Inder Verma, Ph.D., a professor in the Laboratory of Genetics at the Salk Institute and Doctor Belmonte were able to take a diseased cell and convert it into a healthy cell. FA is a genetic blood disorder that can cause cancer and other hematological abnormalities. It can lead to bone marrow failure, leukemia, and other cancers.

The scientists took a hair or skin sample from patients with FA and corrected the defect with gene therapy. They then used the cells to create induced pluripotent stem cells. These cells were indistinguishable from embryonic stem cells.

Although they are much closer to curing genetic diseases, there are some hurdles to overcome. According to the researchers the FA-iPS cells could be the source of transplantable hematopoietic progenitor cells, but iPS cells can cause tumors.

They are now working on the tumor problem. “We haven’t cured a human being, but we have cured a cell,” Belmonte explains. “In theory we could transplant it into a human and cure the disease.”

By Dan Wilson

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