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Curing Beta Thalassemia With Stem Cells and CRISPR

Posted by on Apr 10, 2018 9:00:00 AM


A Phase I clinical trial to test the efficacy of genetically modified autologous (the patient’s own) stem cells to treat beta-thalassemia has been initiated. This condition is an inherited disease that affects the production of hemoglobin, which is responsible for carrying oxygen in the body and delivering it to tissues and vital organs. With thousands of new cases every year, this condition often results in fatigue, bone fragility and extreme anemia (a deficiency of iron in the blood). This trial aims to create a groundbreaking protocol that would obtain autologous stem cells from the patients, genetically alter them to produce the missing protein responsible for the condition and, reintroduce the stem cells back into the body through a transfusion.The trial represents a confluence of two trends that will have a profound, transformative impact on the treatment of genetic disorders – STEM CELLS and CRISPR technology.  Identifying and correcting defective genes [CRISPR] that are the cause of a genetic disorder, coupled with the unique properties of stem cells to proliferate [the use of autologous stem cells eliminates the need to find a donor and removes the risk of rejection] represents a paradigm shift in the treatment of genetic disorders. The treatment seeks to cure the patient of the disease, rather than seeking to minimize the symptoms. The trial will be the first of its kind to be conducted in Europe, and, if successful, can pave the way for a gene altering protocol that would allow for the treatment of virtually any genetic disorder.

“Certainly, 2018 promises to be the big year for clinical trials using CRISPR based genome editing. Results presented by [CRISPR Therapeutics] at a hematology meeting showed that the method dramatically increased fetal hemoglobin in beta thalassemia  patients’ cells. The therapy successfully edited over 90 per cent of blood stem cells removed from patients, which were re-transfused,” comments Helen O’Neill, a geneticist at the University College London.

 Advances in stem cell based regenerative treatments are creating more effective treatment options for patients suffering from a wide variety of disease, trauma and injury. 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.


The Future of Regenerative Medicine is Now™

To view the full article, click here.

Topics: Beta-thalassemia, stem cell therapy, Genetic Diseases, genetic engineering


AAOMS - American Association of Oral and Maxillofacial Surgeons


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