CRISPR - mediated Mutation Correction of Hematopoietic Stem Cells from Patients with X-linked Chronic Granulomatous Disease Using Non-Viral Cell Engineering (maxcyte.com)
Patients with chronic granulomatous disease (CGD) are for example incapable of expressing the functional NOX2 enzyme. (sciencedaily.com)
ST. LOUIS, MISSOURI — Gene therapy has apparently restored the immune systems of two patients with a rare inherited disorder called chronic granulomatous disease (CGD). (sciencemag.org)