From Lab to Life: The Miracle of Gene Repair |
source: depositphotos.com
The heading of Jeff Coller’s New York Times article (April 9) is rather bombastic: “This may be the most important medical story of the decade.”
What is this bombshell story?
Little K.J. Muldoon was born with a fatal genetic ailment. He lacked a gene that generates a crucial compound, ‘carbomyl phosphate synthetase 1’, that enables the nitrogen from protein metabolism to be metabolized. As a result, ammonia NH3 accumulates – and ultimately kills. This is a very rare genetic disease – one in 1.3 million births. It is named after the missing key compound: ........