Rare Disease
April 22, 2026 / April 22, 2026 by Dawn Lam
In May of 2025, a month after the last dose of k-abe was administered, a remarkable paper was published detailing the conception, development and treatment of the infant KJ Muldoon with an in vivo base editor to cure his carbamoyl-phosphate synthetase 1 (CPS1) deficiency.[1] CPS1 deficiency, which affects approximately 1 in 1,300,000 people, is a […]
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