New Genetic Therapy Provides Breakthrough for Down Syndrome

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Every year, 6,000 American babies are born with an extra copy of chromosome 21, the genetic cause of Down Syndrome.

Researchers have puzzled over that extra chromosome since Dr. Jerome Lejeune discovered it in 1959. But this week, doctors at the University of Massachusetts Medical School announced a breakthrough that could have significant implications for how we treat the disease.

According to Dr. Brian Skotko, co-director of the Down Syndrome Program at Massachusetts General Hospital and professor at Harvard Medical School, this landmark study demonstrates that "it is possible, at least in a Petri dish, to be able to turn off that extra chromosome."

"I have to say," Dr. Skotko continues, "over the past decade, we never thought this would be possible. But Dr. Lawrence and her team have shown that, perhaps in the future, it is possible."

Dr. Skotko describes the implications for patients living with Down Syndrome, and the potential ethical issues that could arise from this new genetic therapy. 

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