doctorslounge : declared in Gene Therapy Described in a Patient With Sickle Cell Disease --Doctors Lounge

"Longer follow-up is required to confirm the durability of the efficacy and safety profile observed, and data from additional evaluations of gene therapy in a larger cohort of patients to confirm the promise of gene therapy for sickle cell disease are lacking," the authors write. First, the researchers extracted a stem cell supply from their adolescent patient's bone marrow, before using chemotherapy to wipe out the remaining stem cells. Then they used a modified virus to deliver an "anti-sickling" version of the beta globin gene into the stem cells they'd removed before chemotherapy. The modified stem cells were infused back into the patient. Back to Journal ArticlesGene Therapy Described in a Patient With Sickle Cell DiseaseBut treatment has only been given to one patient so far Share | Comments: (0) Tell-a-FriendTHURSDAY, March 2, 2017 (HealthDay News) -- In a brief report published in the March 2 issue of the New England Journal of Medicine, researchers detail their early success using gene therapy to treat, or even potentially cure, sickle cell disease.

'Not A Cure,' But Cambridge Biotech's Gene Therapy Gets Teen With Sickle Cell Off Meds

"It shows gene therapy is on the right track. Gene therapy offers hope of a lasting one. A French teen who was given gene therapy for sickle cell disease more than two years ago now has enough properly working red blood cells to dodge the effects of the disorder, researchers report. Full results have not been reported, but the gene therapy has not taken hold as well in some of them as it did in the French teen. About 90,000 people in the U.S., mostly blacks, have sickle cell, the first disease for which a molecular cause was found.


Sickle cell disease is one that mainly affects people of African, Caribbean, Middle Eastern, Eastern Mediterranean, and Asian origin. Fifteen months later, the boy, now 15, is not suffering from any of the effects of sickle cell disease. The afflicted have unusually shaped (sickle) blood cells thanks to a coding error in the gene for hemoglobin, the oxygen-carrying segment of red blood cells. Now, the same type of process has been used to cancel out all the symptoms of a French teenager's sickle cell disease. Sickle cells in among normal blood cells.



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