[Gene editing is changing the treatment of hereditary diseases].

Lakartidningen

docent, verksamhetschef, Karolinska centrum för cellterapi (KCC), Karolinska universitetssjukhuset, Stockholm; Karolins-ka ATMP-centrum; institutionen för laboratorie-medicin, Karolinska institutet.

Published: January 2025

Gene editing is a novel technology within gene therapy, which changes sequences in chromosomal DNA with precision. Even if there are alternative strategies, the Nobel Prize-winning CRISPR/Cas technology has become the dominating principle. During recent years base editing and prime editing, permitting editing without DNA double-strand breaks, have been developed. The first clinical gene editing results were reported in 2021; since then many patients have been treated, and recently the first treatment was approved as a novel therapy in the UK and later in USA and in EU. This update describes various aspects including methodological developments and safety.

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