Using a patient’s own stem cells, researchers at Johns Hopkins have corrected the genetic alteration that causes sickle cell disease (SCD), a painful, disabling inherited blood disorder that affects mostly African-Americans. The corrected stem cells were coaxed into immature red blood cells in a test tube that then turned on a normal version of the gene.
The research team cautions that the work, done only in the laboratory, is years away from clinical use in patients, but should provide tools for developing gene therapies for SCD and a variety of other blood disorders.
In an article published online August 31 in
Bond strengthened: Eight-year-old Thamirabharuni, holding her brother who donated the stem cells, did not suffer from rejection or graft versus host disease as the tissue match was perfect – Photo: V. Ganesan
Eight-year-old Thamirabharuni and her one-year-old brother Pugazhendhi share a special kind of bond not commonly seen among siblings. Thanks to her brother, Thamirabharuni no longer suffers from thalassemia disease.
The stem cells transplanted in March helped her get rid of thalassemia. And hundred days after the procedure, one can safely say that her disease has been cured.
The stem cells that were transplanted came from two different sources
Image by steve p2008 via Flickr
After making news on several occasions, scientists may have made a definitive breakthrough, with the first possible transfusion using blood obtained from embryonic stem cells possibly coming within the next three years. The transfusion would be done with type O blood, which can be donated to any patient, and would be obtained by researchers using excess embryos from assisted fertilization. The project, which will be led by Marc Turner of Edinburgh University, will also receive contributions from the Transfusion and Transplant Service of the British National Health Service, as well as the
Scientists and transplant clinicians at the Ansary Stem Cell Institute at Weill Cornell Medical College and the Center for Cell Engineering at Memorial Sloan Kettering Cancer Center have been awarded a $15.7 million, four-year research grant from the New York State Stem Cell Science Program (NYSTEM).
The scientists will translate their approach to manipulate hematopoietic stem cells to cure acquired and inherited blood disorders. For many patients with such blood diseases, including sickle cell disease, the only hope for a cure requires transplanting normal blood stem cells.
But in many instances suitable blood stem cells cannot be found or there are
British scientists have created human red blood cells from spare embryonic stem cells, a major breakthrough they claim could soon pave the way for production of synthetic ‘O-negative‘ blood for medical transfusions.
The red blood cells have been produced from stem cells from spare IVF embryos as part of a three-billion-pound project to develop an alternative source of O-negative blood, the universal donor group which can be transfused into people without fear of rejection, ‘The Independent’ reported.
In their research, the scientists used more than a 100 spare embryos left over from treatment at fertility clinics to establish several embryonic stem