BABY STEPS IN PERSONALISED GENE EDITING

The biggest health story this past week, globally, was about little KJ Muldoon and what doctors could do for him.

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Muldoon, as the world knows him, is a U.S. infant with a rare condition who has become history’s first patient to be treated with a personalised gene-editing technique. Besides raising hopes of cure or treatment for other people with obscure illnesses, the exact process of rehabilitating the nine-and-a-half-month-old KJ itself was simply fascinating. Being witness to medical history can be an upper in itself for some.

Shortly after birth, he was diagnosed with a rare and serious condition called CPS1 deficiency, caused by a mutation in a gene that produces an enzyme key to liver function, and prevents people with it from eliminating certain kinds of toxic waste. With the prognosis grim, either death or a liver transplant, doctors suggested something that had never been done before: a personalised treatment to fix the baby’s genome using what amounts to a pair of molecular scissors -- the technique called Crispr-Cas9, which earned its creators the Nobel prize for chemistry in 2020.