Lentiviral gene therapy for X-linked chronic granulomatous disease.

ABSTRACT: Chronic granulomatous disease (CGD) is a rare inherited disorder of phagocytic cells^1,2. We report the initial results of nine severely affected X-linked CGD (X-CGD) patients who received ex vivo autologous CD34⁺ hematopoietic stem and progenitor cell-based lentiviral gene therapy following myeloablative conditioning in first-in-human studies (trial registry nos. NCT02234934 and NCT01855685). The primary objectives were to assess the safety and evaluate the efficacy and stability of biochemical and functional reconstitution in the progeny of engrafted cells at 12 months. The secondary objectives included the evaluation of augmented immunity against bacterial and fungal infection, as well as assessment of hematopoietic stem cell transduction and engraftment. Two enroll

SUBMITTER: Kohn DB 

PROVIDER: S-EPMC7115833 | biostudies-literature | 2020 Feb

REPOSITORIES: biostudies-literature