Preclinical Optimization and Safety Studies of a New Lentiviral Gene Therapy for p47^phox-Deficient Chronic Granulomatous Disease.

ABSTRACT: Chronic granulomatous disease (CGD) is an inherited blood disorder of phagocytic cells 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 stable correction over time, with no adverse events related to the gene therapy procedure. We have recently developed a parallel lentiviral vector for p47^phox-deficient CGD (p47^phoxCGD), the second most common form of this disease. Using this vector, we have observed biochemical correction of CGD in a mouse model of the disease. In preparation for clinical trial approval, we have performed standardized preclinical studies following Good Laboratory Practice (GLP) principles, to assess the safety of the gene

SUBMITTER: Schejtman A 

PROVIDER: S-EPMC8575060 | biostudies-literature | 2021 Sep

REPOSITORIES: biostudies-literature