Gene therapy possibilities for curing in next generation
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The future of medicine is bright. This year brought several first in class technologies over the finish line. The underpinning of these radical medical developments is the understanding of how human genes can be modified to cure diseases and save lives. The technology which captured the most attention this year is CAR-T therapy. A CAR-T therapy consists of the infusion of engineered T cells that express a Chimeric Antigen Receptor (CAR) on their cell membrane that targets other blood cells that may be malignant. To deliver the therapy, white blood cells are removed from the body, genetically altered with the cancer targeting CAR, and replaced back into the bloodstream. Those modified white blood cells flow through the body chewing up B cells (and cancer within that B cell population) with an efficiency and safety profile unprecedented in previous treatments. These results are partially thanks to a genetic delivery “vector” called Lentivirus. CAR-T may have been one of the first gene therapies to win the approval of the FDA, but there will soon be a lot more. Today there are more than 800 ongoing gene therapy clinical trials. Shortly after Kymriah (the first CAR-T) won approval, Yescarta (another CAR-T) followed it. Those approvals will be remembered as some of the drips before the dam broke releasing a plethora of new, highly-effective gene and cell therapies