Second patient added to human gene-editing study, with no side effects so far

| | February 13, 2018

A second patient has been treated in a historic gene editing study in California, and no major side effects or safety issues have emerged from the first man’s treatment nearly three months ago, doctors revealed [February 6].

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In November, 44-year-old Brian Madeux became the first person to have gene editing inside the body for a metabolic disease called Hunter syndrome that’s caused by a bad gene. Through an IV, he received many copies of a corrective gene and a genetic tool to put it in a precise spot in his DNA.

Madeux had dizziness, cold sweats and weakness four days after the treatment but they went away on their own in a day, Harmatz said. Madeux also had a severe cough and a partially collapsed lung, but these were deemed unrelated to the gene therapy because he had had similar problems previously. Importantly, there were no signs of harm to his liver.

A prominent gene therapy scientist, Dr. James Wilson of the University of Pennsylvania, published two studies reporting liver and other serious problems in monkeys and piglets that were given experimental gene therapies. Several had to be euthanized.

An editorial in the journal Human Gene Therapy, which published one of Wilson’s animal studies, said gene therapy experiments should not stop because that might deprive patients of potentially life-saving treatments.

Read full, original post: 2nd man has gene editing; therapy has no safety flags so far

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