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2017 was the year of gene-therapy breakthroughs

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Researchers announced more miraculous cures of patients with rare and life-threatening diseases who were treated with experimental therapies. Last year, we wrote that 2016 was gene therapy’s most promising year.

Scientists removed stem cells from the boy’s bone marrow and modified them in the lab by introducing copies of a gene to prevent his red blood cells from becoming “Sickled.” When the treated cells were infused back into his body, they began to make normal blood cells.

More than two years after treatment, the patient has enough normal red blood cells to evade any side effects of the disorder. Cancer killers This year the FDA approved two pioneering treatments, Kymriah and Yescarta, that use a patient’s own immune cells to fight rare types of cancer. Called CAR-T therapies, these “Living drugs” are made by extracting T cells from patients and genetically engineering them to go after and destroy cancer cells.

A year and a half after treatment, patients have had fewer bleeding issues and have been able to cut back on infusions of clotting factor.


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Post Author: Ethan Siegel

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