Aggregated News
Three years ago, at the age of 50, I found out I have dangerous genes. So in June, I went to Cambridge, Massachusetts, to see how they could be fixed. Among my 6 billion base pairs of DNA are two mutations — one inherited from my father, one from my mother — that give me Alpha-1-antitrypsin deficiency syndrome, also known as AATD or just Alpha-1. My mutation creates a misfolded protein that can’t fight off everyday lung damage, as it normally would. This protein can also pool in the liver, causing degeneration there too. The worst form of the mutation can make a carrier 10 to 20 times more likely to develop emphysema.
I went to Cambridge to visit Beam Therapeutics, which was founded to develop base-editing therapies. This is one of four techniques, three of them built on the science of Crispr, that are now available to permanently alter our DNA. One of the treatments Beam is working on seeks to repair the mutation that causes Alpha-1. In an ongoing trial, 29 volunteers have received the therapy...