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Curing Beta Thalassemia With Stem Cells and CRISPR

Posted by barb@stemsave.com 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.

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

Stem Cell Clinical Trials to Treat Genetic Disorders Announced

Posted by pamela@stemsave.com on Aug 1, 2013 8:03:20 AM

Gene therapies utilizing stem cells are being developed that may lead to a cure for several genetic diseases. Currently, two clinical trials were announced to treat Cerebral Adrenoleukodystrophy (CCALD) and beta-thalassemia/sickle cell disease.

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Topics: sickle cell disease, Phase III, Cerebral Adrenoleukodystrophy, clinical trials, Beta-thalassemia, Stemcells, translational genomics, autologousstemcells, young stem cells, stemsaveblog

AAOMS

AAOMS - American Association of Oral and Maxillofacial Surgeons

 

As the industry leader, StemSave is the only stem cell banking service to be designated as an ASI approved program.

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