Thomas R. Bauer Jr., Ph.D.
Thomas R. Bauer Jr., Ph.D.
Staff Scientist

Center for Cancer Research
National Cancer Institute

Bldg 10-CRC, Rm 3-3264
Bethesda, MD 20892-1203
301-435-7125

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.

Areas of Expertise
CLAD, LAD

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.

Selected Publications
  1. Bauer, Jr. TR, Olson EM, Huo Y, Tuschong LM, Allen JM, Li Y, Burkholder TH, Russell DW.
    Gene therapy. 18: 553-9, 2011. [ Journal Article ]
  2. Hunter MJ, Tuschong LM, Fowler CJ, Bauer, Jr. TR, Burkholder TH, Hickstein DD.
    Molecular Therapy. 19: 113-21, 2011. [ Journal Article ]
  3. Colmont CS, Benketah A, Reed SH, Hawk NV, Telford WG, Ohyama M, Udey MC, Yee CL, Vogel JC, Patel GK.
    Proc. Natl. Acad. Sci. U.S.A.. 110: 1434-9, 2013. [ Journal Article ]
  4. Kanevskiy LM, Telford WG, Sapozhnikov AM, Kovalenko EI.
    Front Immunol. 4: 11, 2013. [ Journal Article ]
  5. Telford WG.
    Methods Mol. Biol.. 946: 163-79, 2013. [ Journal Article ]

Born in Southwestern PA, but raised during my formative years in the suburbs of Los Angeles, I left SoCal for Indiana, where I received a B.S. in Microbiology from the University of Notre Dame in 1985. Desiring warmer weather, I went to the University of Miami (Florida), where I received a Ph.D. in Microbiology and Immunology in 1992 studying human immunoglobulin lambda light chain genes. From there, I joined the laboratory of Dennis Hickstein in Seattle, where I was a postdoc and later acting instructor, working on LAD and CD18, and to my current position as Staff Scientist at the NIH.

Summary

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.

Areas of Expertise
CLAD, LAD

Research

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.

Publications

Selected Publications
  1. Bauer, Jr. TR, Olson EM, Huo Y, Tuschong LM, Allen JM, Li Y, Burkholder TH, Russell DW.
    Gene therapy. 18: 553-9, 2011. [ Journal Article ]
  2. Hunter MJ, Tuschong LM, Fowler CJ, Bauer, Jr. TR, Burkholder TH, Hickstein DD.
    Molecular Therapy. 19: 113-21, 2011. [ Journal Article ]
  3. Colmont CS, Benketah A, Reed SH, Hawk NV, Telford WG, Ohyama M, Udey MC, Yee CL, Vogel JC, Patel GK.
    Proc. Natl. Acad. Sci. U.S.A.. 110: 1434-9, 2013. [ Journal Article ]
  4. Kanevskiy LM, Telford WG, Sapozhnikov AM, Kovalenko EI.
    Front Immunol. 4: 11, 2013. [ Journal Article ]
  5. Telford WG.
    Methods Mol. Biol.. 946: 163-79, 2013. [ Journal Article ]

Biography

Born in Southwestern PA, but raised during my formative years in the suburbs of Los Angeles, I left SoCal for Indiana, where I received a B.S. in Microbiology from the University of Notre Dame in 1985. Desiring warmer weather, I went to the University of Miami (Florida), where I received a Ph.D. in Microbiology and Immunology in 1992 studying human immunoglobulin lambda light chain genes. From there, I joined the laboratory of Dennis Hickstein in Seattle, where I was a postdoc and later acting instructor, working on LAD and CD18, and to my current position as Staff Scientist at the NIH.