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.
In an animal model, a single injection of the modified stem cells , corrected the disorder and persisted in the body for nearly a year. according to Dr. Suvasini Ramaswamy, first author of the study, “The appeal of a cell-based approach is that you minimize the number of treatments that a patient needs…Rather than constant injections, you can do this in one shot." Additionally, this autologous treatment offers the advantage of a localized repair as well as eliminating the risk of rejection and the need for immunosuppression.”
This research protocol could be expanded to treat a variety of genetic disorders utilizing stem cells, correcting malfunctioning genes for long-term cures rather than temporary treatments that focus on the symptoms . To learn more about stem cells, and how families can bank their own valuable stem cells by recovering the very powerful dental pulp stem cells during routine dental procedures; such as wisdom teeth extractions or the during the loss of baby teeth, visit StemSave or call 877-783-6728 (877-StemSave) today.
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