In a landmark moment for genetic medicine, researchers have reported the first peer-reviewed clinical use of prime editing to treat human patients. The New England Journal of Medicine article describes PM359, an autologous hematopoietic stem-cell (HSC) therapy that aims to correct the deletion of two base pairs, a guanine followed by a thymine (delGC), in NCF1 , which is the primary cause of p47phox-deficient chronic granulomatous disease (p47-CGD). Both patients treated in this first-in-human study remained free of new CGD-related complications during the first few months of follow-up.

Two bases, two patients, two successes

CGD is a rare inherited immunodeficiency marked by defective NADPH oxidase activity, resulting in recurrent bacterial and fungal infections and chr

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