June 01, 2009
Gene Therapy And Induced Pluripotent Stem Cells Against Fanconi Anemia

Salk researchers did genetic repair on cells from patients with Fanconi anemia and then turned the cells into pluripotent stem cells and then turned the cells into the type of cells that make red blood cells.

LA JOLLA, CAA study led by researchers at the Salk Institute for Biological Studies, has catapulted the field of regenerative medicine significantly forward, proving in principle that a human genetic disease can be cured using a combination of gene therapy and induced pluripotent stem (iPS) cell technology. The study, published in the May 31, 2009 early online edition of Nature, is a major milestone on the path from the laboratory to the clinic.

The probably have safety concerns that hold back their injecting the resulting cells back into patients.

After taking hair or skin cells from patients with Fanconi anemia, the investigators corrected the defective gene in the patients' cells using gene therapy techniques pioneered in Verma's laboratory. They then successfully reprogrammed the repaired cells into induced pluripotent stem (iPS) cells using a combination of transcription factors, OCT4, SOX2, KLF4 and cMYC. The resulting FA-iPS cells were indistinguishable from human embryonic stem cells and iPS cells generated from healthy donors.

Since bone marrow failure as a result of the progressive decline in the numbers of functional hematopoietic stem cells is the most prominent feature of Fanconi anemia, the researchers then tested whether patient-specific iPS cells could be used as a source for transplantable hematopoietic stem cells. They found that FA-iPS cells readily differentiated into hematopoietic progenitor cells primed to differentiate into healthy blood cells.

"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."

The development of therapies to repair genetic diseases involves developing capabilities needed for rejuvenation of old bodies. Cells malfunction due to aging just as they malfunction due to harmful genetic mutations inherited at birth. Every gene therapy and cell therapy developed to treat genetic diseases puts us closer to treatments to reverse damage of an aged body.

Rejuvenation therapies will first get developed as a side benefit to the development of therapies to treat genetic diseases and other diseases that are not the result of aging. Therefore the development of rejuvenation therapies is inevitable.

Share |      Randall Parker, 2009 June 01 11:53 PM  Biotech Stem Cells

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