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Doctors reverse teen's sickle cell disease with innovative gene therapy

Posted on: 5 March 2017, source: normangeestar.net
A 16-year-old boy in France has become the first person ever to be successfully treated with a new first-of-its-kind gene therapy for sickle cell disease. Doctors removed the teen's bone marrow and genetically altered it in the laboratory to correct the defect in his DNA that causes sickle cell disease. The study stated 15 months after the very first treatment that the boy who once had significant internal damage - so much so that it led to a removed spleen and a hip replacement - now has no sign of the disease.


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The patient's sickle cell was so severe that the doctors chose to try out a gene therapy that re-engineered his extracted bone marrow's DNA to create the right type of red blood cells. Still, the possibility of a life without medication and treatment will be equally helpful to the millions around the world living with this disease. In the case of sickle cell disease, hemoglobin, a protein in red blood cells that carries oxygen throughout the body, is misshapen. He visits the hospital every month to have blood transfusion so that the defective blood will be diluted. A virus was used to infect the bone marrow with new, correct instructions. With the new instructions, the bone marrow was then put back into the patient. He experiences neither pain nor hospitalization, and does not require blood transfusion as well. "But of course we need to perform the same therapy in many patients to feel confident that it is robust enough to propose it as a mainstream therapy".

"The successful outcome in Patient 1204 demonstrates the promise of treatment with LentiGlobin gene therapy in patients with severe [sickle cell disease] and serves as a guide for our efforts to optimize outcomes in future patients", said Dr. David Davidson, the company's chief medical officer, in a statement. But for now, the gene therapy technique has a compelling proof-of-concept. The next big challenge will be to transform this pioneering science into something that really can help millions of people. Though doctors are still hesitant to call the therapy a cure for sickle cell disease, it is a major step toward combating the disease. Individuals with sickle cell disease have red blood cells that are sickle or crescent-shaped instead of the usual, healthy round shape.

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Posted on: 5 March 2017, source: normangeestar.net
A 16-year-old boy in France has become the first person ever to be successfully treated with a new first-of-its-kind gene therapy for sickle cell disease. Doctors removed the teen's bone marrow and genetically altered it in the laboratory to correct the defect in his DNA that causes sickle cell disease. The study stated 15 months after the very first treatment that the boy who once had significant internal damage - so much so that it led to a removed spleen and a hip replacement - now has no sign of the disease.
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