Using genome editing to cure genetic diseases may no longer just be in science fictions. Recently, the first industry sponsored CRISPR treatment for patients with genetic diseases is going to be tested in human. A company based in Massachusetts, USA., CRISPR Therapeutics, has submitted an application to the European Medicines Agency for permission to launch Phase I/II clinical trials using CRISPR treatment in human. The proposed gene editing therapy, CTX001, is going to be tested in patients with sickle cell disease or β thalassemia, two common blood disorders in Europe and the US.
By genetically editing hematopoietic stem cells harvested from patients ex vivo using CRISPR technology, researchers are able to consistently edit more than 80% of these cells. Transferring these genetic modified hematopoietic stem cells back into mouse models resulted in significant increases in the amount of red blood cells with high levels of fetal hemoglobin (HbF). Thus, can be used to treat genetic disorders with impaired hemoglobin metabolism. Now, these clinical studies will further test whether this therapy, CTX001, will have the same effects in human.
If these clinical trials show positive results, more clinical studies targeting different genetic disorders using CRISPR genome editing will be encouraged. Having a tool/treatment that is able to correct all genetic diseases by fixing the root of these disorders in human may no longer be just an science fiction fantasy. Despite all the ethical debates this treatment has brought out, using genome editing technics to edit ourselves may just be around the corner.
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