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Stem Cell and Gene Therapy Will Cure Genetic Diseases?

A new five-year $6.7-million research project will seek for a cure for life-threatening genetic disorders like sickle cell anemia. The study will be carried out at University of California, San-Francisco (UCSF), the lucky winner of the grant from the National Institutes of Health (NIH).

The new approach to treating genetic diseases that will be studied will include gene and stem cell therapy. The research will be based on adult—not embryonic—stem cells. First the researchers plan to gather patients' own blood cells and convert them to induced pluripotent stem cells. These cells carry genetic error, which the researchers plan to find and eliminate through experimental genetic engineering techniques. Instead, the correct portion of the DNA will be placed into the cells.

After that, the researchers plan to convert the repaired induced pluripotent stem cells into more specialized stem cells, namely, hematopoietic stem cells. These cells are to be transplanted back to the patient and are expected to give rise to properly functioning blood cells as well as restore immune system components.

With this approach involving autologous—patients own—stem cells, the researchers aim to leave behind issues of donor-recipient matching and eliminate the risk of graft versus host disease—the issues brought by bone marrow transplantation.

The team plans to work with beta thalassemia and sickle cell anemia patients whose only hope now is bone marrow or cord blood transplant from a compatible donor. Both these diseases are caused by a single gene defect that encodes beta globin, a part of hemoglobin which is the component of red stem cells responsible for oxygen transportation in the body. The researchers hope that both adults and children will benefit from the new method.