Lentiviral gene therapy rescues p47phox chronic granulomatous disease and the ability to fight Salmonella infection in mice
Sign Up to like & getrecommendations! Published in 2020 at "Gene Therapy"
DOI: 10.1038/s41434-020-0164-6
Abstract: Chronic granulomatous disease (CGD) is an inherited primary immunodeficiency disorder characterised by recurrent and often life-threatening infections and hyperinflammation. It is caused by defects of the phagocytic NADPH oxidase, a multicomponent enzyme system responsible for… read more here.
Keywords: granulomatous disease; chronic granulomatous; gene therapy; lentiviral gene ... See more keywords
Pre-clinical optimisation and safety studies of a new lentiviral gene therapy for p47phox-deficient chronic granulomatous disease.
Sign Up to like & getrecommendations! Published in 2021 at "Human gene therapy"
DOI: 10.1089/hum.2020.276
Abstract: Chronic granulomatous disease (CGD) is an inherited blood disorder that renders patients susceptible to infections and inflammation. A recent clinical trial of lentiviral gene therapy for the most frequent form of CGD, X-linked, has demonstrated… read more here.
Keywords: chronic granulomatous; gene therapy; disease; lentiviral gene ... See more keywords
Lentiviral Gene Therapy for X-Linked Chronic Granulomatous Disease
Sign Up to like & getrecommendations! Published in 2020 at "Pediatrics"
DOI: 10.1542/peds.2020-023861rrrr
Abstract: DB Kohn, C Booth, EM Kang. Nature Med. 2020;26(2):200–206 To assess the safety and evaluate the efficacy and stability of ex vivo autologous CD34+ hematopoietic stem and progenitor cell based lentiviral gene therapy for X-linked… read more here.
Keywords: linked chronic; lentiviral gene; gene therapy; therapy linked ... See more keywords