Rare Disease Industry Insights
April 22, 2026 / April 23, 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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Oligo Modification By Application
March 23, 2026 / April 9, 2026 by Dawn Lam
Background Ligases are ubiquitous. They are found in every living organism and are required for cell division – stitching together the Okazaki fragments produced during DNA replication and maintaining genetic integrity by performing the final step in every DNA repair pathway – the crucial ligation step – which is the formation of a phosphodiester linkage […]
