Gene editing has reshaped biology and produced its first approved medicine, yet CRISPR, the widely used gene-editing technology, faces two stubborn obstacles on its march into the clinic: the leading editors are too big to deliver with standard viral vectors, and they can cut DNA in the wrong place, raising fears about unintended genetic changes.

Two new studies from researchers in KAUST describe a new generation of genome editors designed to tackle both challenges at once. These editors are small enough to fit inside a single viral delivery vehicle, while also engineered to edit DNA with greater precision.

Currently, all approved CRISPR therapies require cells to be removed from patients, edited outside the body, and reinfused after intensive chemotherapy. This complex procedure can cost millions of dollars, carry substantial treatment-related risks and work only for conditions such as sickle-cell disease and beta-thalassemia that originate in blood-forming stem cells.

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