A 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.
A team for the Salk Institute in the USA and colleague at the CMRB, and the CIEMAT in Madrid, Spain, decided to focus on Fanconi anemia (FA), a genetic disorder responsible for a series of hematological abnormalities that impair the body's ability to fight infection, deliver oxygen, and clot blood. Caused by mutations in one of 13 Fanconi anemia (FA) genes, the disease often leads to bone marrow failure, leukemia, and other cancers.
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
In an interview reported by Health News Track one of the leading members of the team stressed that research at this stage was limited to the laboratory, stating "We haven't cured a human being, but we have cured a cell,". Definitive proof of the efficacy of this approach must await transplanting the cells into patients.
The abstract of the Nature publication is available here.
0 comments:
Post a Comment