The New England journal of medicine
Prime Editing for p47phox-Deficient Chronic Granulomatous Disease.
Jennifer L Gori, Elie Haddad, Haydar Frangoul, Donald B Kohn, Emma C Morris, Bradley N Martin, Briana A Deary, McKinley Nickerson, Rebecca L Scholz, Isabel Fernandez, Karine Leveille, Suk See De Ravin, Elizabeth M Kang, Marie Pierzynski, Tyra Estwick
Published: 202510.1056/NEJMoa2509807
Abstract
Chronic granulomatous disease (CGD) is a severe monogenic immunodeficiency caused by damaging variants in genes required for microbicidal NADPH oxidase activity. Autosomal recessive p47phox-deficient CGD (p47-CGD) is predominantly caused by a two-nuc…
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