How Does Gene Therapy Work?04:33

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Published on June 1, 2017

Scientists have promised that gene therapy will be the next big leap for medicine. It’s a term that’s tossed about regularly, but what is it exactly? Trace shows us how scientists can change your very genetic code.

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How does gene therapy work?

“Gene therapy is designed to introduce genetic material into cells to compensate for abnormal genes or to make a beneficial protein. If a mutated gene causes a necessary protein to be faulty or missing, gene therapy may be able to introduce a normal copy of the gene to restore the function of the protein.”

Gene therapy trial ‘cures children’

“A disease which robs children of the ability to walk and talk has been cured by pioneering gene therapy to correct errors in their DNA, say doctors.”

Gene therapy cures diabetic dogs

“Five diabetic beagles no longer needed insulin injections after being given two extra genes, with two of them still alive more than four years later.”

Gene Therapy for Cancer: Questions and Answers

“Gene therapy is an experimental treatment that involves introducing genetic material into a person’s cells to fight or prevent disease.”

How does gene therapy work?

“Gene therapy is the addition of new genes to a patient’s cells to replace missing or malfunctioning genes. Researchers typically do this using a virus to carry the genetic cargo into cells, because that’s what viruses evolved to do with their own genetic material.”

Gene therapy cures leukaemia in eight days

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WITHIN just eight days of starting a novel gene therapy, David Aponte’s “incurable” leukaemia had vanished. For four other patients, the same happened within eight weeks, although one later died from a blood clot unrelated to the treatment, and another after relapsing.

Cell Therapy Shows Promise for Acute Type of Leukemia

“A treatment that genetically alters a patient’s own immune cells to fight cancer has, for the first time, produced remissions in adults with an acute leukemia that is usually lethal, researchers are reporting.”

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