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Thomas R. Bauer Jr., Ph.D.
For over a decade, our lab has been working towards gene therapy of the childhood immunodeficiency disease 'Leukocyte Adhesion Deficiency', also known as LAD. Patients with LAD have defects in their leukocyte integrin CD18 gene, which leads to defective leukocytes unable to adhere to and migrate to sites of bacterial infection. Although hematopoietic stem cell transplantation can cure LAD patients, too few patients have suitable transplantation donors. We have investigated the possibility of transferring a functional copy of the CD18 gene into patients' hematopoietic stem cells using gene transfer with retroviral vectors. Towards this end, we have utilized an animal model of LAD in dogs, know as 'canine Leukocyte Adhesion Deficiency' or CLAD to investigate the various vectors and conditions required for successful gene transfer. We have shown that by using gene transfer of the canine CD18, we are able to correct the CD18 defect in canine leukocytes both in vitro and in vivo, leading to dogs free of their CLAD disease.
This page was last updated on 2/21/2013.