Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1.

ABSTRACT:

Background

Allogeneic hematopoietic stem-cell transplantation for X-linked severe combined immunodeficiency (SCID-X1) often fails to reconstitute immunity associated with T cells, B cells, and natural killer (NK) cells when matched sibling donors are unavailable unless high-dose chemotherapy is given. In previous studies, autologous gene therapy with γ-retroviral vectors failed to reconstitute B-cell and NK-cell immunity and was complicated by vector-related leukemia.

Methods

We performed a dual-center, phase 1-2 safety and efficacy study of a lentiviral vector to transfer IL2RG complementary DNA to bone marrow stem cells after low-exposure, targeted busulfan conditioning in eight infants with newly diagnosed SCID-X1.

Results

Eight infants with SCID-X1 were fol

SUBMITTER: Mamcarz E 

PROVIDER: S-EPMC6636624 | biostudies-literature | 2019 Apr

REPOSITORIES: biostudies-literature