Industry Insights Rare Disease
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 […]
Oligo Modification By Application CRISPR
June 5, 2024 / February 5, 2025 by Dawn Lam
What is CRISPR Technology? CRISPR/CasX Technology (Clustered Regularly Interspaced Short Palindromic Repeats), derived from bacteria, has been a cornerstone of genetic engineering in the therapeutic industry for decades. The technology is versatile with its application, spanning across various fields, from agriculture to medicine, and enabling scientists to modify DNA sequences with remarkable accuracy. The most […]
