Gene therapy for sickle cell disease may help improve a major contributing factor to stroke risk in patients, reports a new study from St. Jude Children's Research Hospital. Many people with sickle cell disease experience increased brain ischemia, where oxygen is not delivered properly to brain tissues, potentially leading to strokes. A part of the risk for these events comes from increased blood flow speed in the brain. Findings from three patients in a gene therapy clinical trial showed that gene therapy treatment significantly improved blood flow in the brain. These results demonstrate that people with these risk factors may benefit from gene therapy and should be considered for future clinical trials of gene therapy. The findings were published in the American Journal of Hematology.
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