Monday, November 20, 2023

Sickle Cell Gene Therapy May Cause Cancer Linked Mutations

 There are forms of gene therapy that has the potential to benefit patients with genetic blood disorders. There is a study on individuals in a trial for sickle cell disease that suggests  key process within giving out the treatment might cause mutation that turn blood stem cells cancerous. In a very recent study that was lead by the University of York reported that  six patients involved in treatment for sickle cell gene therapy had showed signs of mutations that are linked with the development of cancer. Scientists responsible for the study says that this case needs more of a long-term study so they can understand and determine how the cells continue to evolve. Sickle Cell disease is where an inherited disorder in which causes pathological changes to an individual's hemoglobin. This is where the disease causes red blood cells to harden and obtain the "sickle" shape and die. With this, gene therapy can alter an individual's genes in a certain way that corrects the mutation or makes they can express a different, healthy form of hemoglobin. 

    Researchers paired whole-genome sequencing with new methods to follow and compare the DNA of blood stem cells in patients before and also after they undergo their treatment. After the study they were able to find that there were numerous driver mutations that have been linked to blood cancer. The researchers also describe that the treatment itself is not causing the mutations, but they say it appears to come from one of the earlier parts of the process. As described in the beginning they would need to undergo further research and study and identify how the cells evolve like that. 


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