Preclinical safety and efficacy of lentiviral-mediated gene therapy for leukocyte adhesion deficiency type I.

Leukocyte adhesion deficiency type I (LAD-I) is a primary immunodeficiency caused by mutations in the gene, which encodes for the CD18 subunit of β-integrins. Deficient expression of β-integrins results in impaired neutrophil migration in response to bacterial and fungal infections. Using a lentiviral vector (LV) that mediates a preferential myeloid expression of human CD18 (Chim.

The downregulated membrane expression of CD18 in CD34 cells defines a primitive population of human hematopoietic stem cells.

Background: CD18 is the common beta subunit of β integrins, which are expressed on hematopoietic cells. β integrins are essential for cell adhesion and leukocyte trafficking.

Methods: Here we have analyzed the expression of CD18 in different subsets of human hematopoietic stem and progenitor cells (HSPCs) from cord blood (CB), bone marrow (BM), and mobilized peripheral blood (mPB) samples.