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02 Dec
Researchers Make Progress in Treating Genetic Diseases Researchers Make Progress in Treating Genetic Diseases

Researchers Make Progress in Treating Genetic Diseases

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Over the years, Brian Madeux has had 26 surgeries to minimize symptoms of Hunter Syndrome, a serious genetic disorder with a general life expectancy of 15 years or less. But with no known cure available, the 44-year-old has been forced to live with the disease, which gradually damages various areas of the body, including thickening the walls of the heart.

In early November, Madeux was part of a revolutionary experiment in genetic therapy. For the first time, a gene will be inserted into the human body with the express purpose of altering a person’s DNA. The procedure is not guaranteed to work. In fact, the gene must be installed in a very specific part of the body to be effective. If misplaced, the change will be permanent and unfortunately, doctors likely won’t know for a few months whether the gene-editing procedure was successful.

Sangamo Therapeutics is in charge of the clinical trial, which corrects the gene Madeux has been missing since birth. The company is heavily invested in precise gene-editing therapy, also currently recruiting for trials in patients with hemophilia B and Hurler Syndrome. If the trial is successful, it could be revolutionary in moving gene therapy forward.

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Read 939 times Last modified on Monday, 20 November 2017 07:54
Stephanie Faris

Stephanie is a freelance writer and novelist whose work has appeared on NYPost.com, PSMag.com, the Intuit Small Business Blog, and many others. She is the Simon & Schuster author of 8 children's books, including the Piper Morgan chapter book series, 25 Roses, and 30 Days of No Gossip.

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