Genome engineering
(The Boston Globe, November 25, 2013)
When a tumor grows on an organ, doctors remove it. When a hip stops working, they replace it. When a faulty gene causes a disorder such as Huntington’s disease or sickle cell anemia, however, there isn’t a surgery to fix the problem. A new Cambridge life-science company aims to develop therapies that can put troublesome genes under the knife, so to speak, cutting out bad DNA like a scalpel excises bad tissue. The company is backed by a $43 million initial investment by three well-known venture capital firm. The company, which hopes to eventually treat people with genetic disorders for which there are few or no options today, is embarking on its venture with an almost unbridled optimism that is rare in medicine.