Company claims signs of success with CRISPR-edited stem cell transplants for two genetic diseases

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Medical teams and researchers started using CRISPR to rectify medical conditions. CRISPR, based in Zug, Switzerland, announced last week that 2 patients benefited from intervention using CRISPR. One patient had sickle cell anemia and the second patient had beta thalassemia.

Prior to this new treatment, practitioners gave red blood cells regularly with multiple infusions throughout the year. In fact, Vertex Pharmaceuticals collaborated with CRISPR Therapeutics to block/remove a gene that prevents the creation of fetal hemoglobin. Furthermore, both patients generated high levels of fetal hemoglobin and didn’t need more transfusions after the stem cell transplant that included chemotherapy. Besides, the benefits were still visible 9 months after the treatment.

“These early findings are a triumph for patients, CRISPR, and genetics,” Bruce Conklin, a clinician who focuses on stem cell treatments of cardiovascular disease at the University of California, San Francisco, told Science after reviewing the limited data the companies provided in the call and a press release. “They chose patients with severe versions of the disease and the results are truly remarkable.” Moreover, other companies are working on competing gene therapy and gene-editing technologies for the same disorders. Unfortunately, no further data was made available to media or other outlets for questions and comments. Read the full article to see the link on the STAT reports.

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