Vectorology - GEG Tech top picks

Leukocyte Adhesion Deficiency Type I (LAD-I) is a primary immunodeficiency caused by mutations in the ITGB2 gene, and is characterized by recurrent and life-threatening bacterial infections. In this study, the authors used three different lentiviral vectors conferring ubiquitous or preferential expression of CD18 in myeloid cells. All vectors induced stable hCD18 expression in hematopoietic cells from mice with a hypomorphic Itgb2 mutation (CD18HYP), both in vitro and in vivo after transplantation of corrected cells into primary and secondary CD18HYP recipients. hCD18+ hematopoietic cells from transplanted CD18HYP mice also showed restoration of mCD11a surface co-expression. Furthermore, these vectors were able to correct the phenotype of human myeloid cells derived from CD34+ progenitors defective in ITGB2 expression. These results support for the first time the use of hCD18-LVs for the treatment of LAD-I patients in clinical trials.