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Hemophilia Cure On The Horizon, Utilizing Stem Cells and CRISPR

Posted by pamela@stemsave.com on Apr 21, 2018 1:01:00 PM

Researchers at the Salk Institute are developing an autologous stem cell cure to treat hemophilia, a genetic disorder affecting millions worldwide. Hemophilia is a disorder in which a person’s blood has a diminished ability to clot, posing the risk of severe bleeding from minor injuries like nosebleeds. Additionally, people with hemophilia are at an even greater risk for internal bleeding, which can arise from minor injuries. Hemophilia is typically inherited but can also be acquired in adulthood. The genetic disorder is caused by an inappropriate immune response where immune cells attack the blood’s clotting factors, or a mutation that prevents the production of the clotting factor altogether. This treatment involves obtaining autologous (the patient’s own) stem cells, editing them to correct the faulty gene with the help of CRISPR (a gene editing technology), and reintroducing the cells back into the body.

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Topics: hemophilia, genetic engineering, CRISPR

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AAOMS - American Association of Oral and Maxillofacial Surgeons

 

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