CURRICULUM VITAE THOMAS ROY BAUER, JR., PH.D. Experimental Transplantation and Immunology Branch Center for Cancer Research National Cancer Institute 10 Center Drive, 10-CRC, 3 East, Room 3-3264 Bethesda, MD 20892-1203 email: bauert@mail.nih.gov Tel: 301-435-7125 Fax: 301-402-5054 Year of Birth 1962 Place of Birth Pennsylvania Citizenship USA Marital Status Single Languages English Education B.S., University of Notre Dame, IN, 1985 (Microbiology) Ph.D., University of Miami, FL,1992 (Microbiology and Immunology) Professional Training and Academic Experience 1992-1996 Fellow in Hematology, Department of Medicine, University of Washington, Seattle, Washington 1996-1998 Senior Fellow in Oncology, Department of Medicine, University of Washington, Seattle, Washington 1998-2001 Acting Instructor, Division of Oncology, Department of Medicine,University of Washington, Seattle, Washington 2001- Staff Scientist, Experimental Transplantation and Immunology, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD Review Boards / Committees Member, Biosafety Committee, VA Puget Sound Health Care System, Seattle, WA 2000 Scientific Review Board, National Gene Vector Laboratories 2002-2007 Honors and Awards Notre Dame Scholar, University of Notre Dame, 1981-1985 Professional Societies American Association for the Advancement of Science (Member) American Society of Gene Therapy (Member) American Society of Hematology (Member) Grant Funding National Institute of Health, Individual National Research Service Award, Post-doctoral fellowship, "Gene Transfer Studies in Leukocyte Adherence Deficiency", 1993-1995 Cystic Fibrosis Foundation, Pilot Project "Transduction of CLAD hematopoietic cells using Foamy virus vectors containing the canine CD18 gene", $50,000/year, 2000. BIBLIOGRAPHY Scholarly Works Submitted Ph.D. Thesis (University of Miami, Miami, FL 1992) Human immunoglobulin lambda light chain genes and lambda-like genes: Identification and characterization of a new C lambda gene and chromosomal localization and expression of human lambda-like genes in pre-B lymphocytes. Journal Articles 1. Bauer, Jr. T.R. and B. Blomberg. 1991. The Human ( L chain Ig Locus: Recharacterization of JC ( 6 and Identification of a Functional JC ( 7. J. Immunology 146: 2813-2820. 2. Bauer, Jr. T.R., H.E. McDermid, M.L. Budarf, M.L. Van Keuren, and B.B. Blomberg. 1993. Physical location of the human lambda-like genes, 14.1, 16.1, and 16.2. Immunogenetics 38: 387-399. 3. Back, A.L., M. Kerkering, D. Baker, T.R Bauer, L.J. Embree, and D.D. Hickstein, D.D. 1993. A point mutation associated with leukocyte adhesion deficiency type 1 of moderate severity. Biochem. Biophys. Res. Commun. 193: 912-8. 4. Bauer, Jr. T.R., W.R.A. Osborne, W.W. Kwok, and D.D. Hickstein, 1994. Expression from leukocyte integrin promoters in retroviral vectors. Hum. Gene Ther. 5, 709-716. 5. Bauer, Jr. T.R., A.D. Miller, and D.D. Hickstein. 1995. Improved Transfer of the Leukocyte Integrin CD18 subunit into Hematopoietic Cell lines by using Retroviral Vectors having a Gibbon Ape Leukemia Virus Envelope. Blood 86: 2379-2387. 6. Bauer, Jr. T.R., and D.D. Hickstein. 1997. Transduction of Human Hematopoietic Cells and Cell Lines using a Retroviral Vector Containing a Modified Murine CD4 Reporter Gene. Hum. Gene Ther. 8: 243-252. 7. Malech, H.L., T.R. Bauer, Jr , and D.D. Hickstein. 1997. Prospects for Gene Therapy of Neutrophil Defects. Sem. Hematol. 34: 355-361. 8. Bauer, Jr., T.R., B.R. Schwartz, W.C. Liles, H.D. Ochs, and D.D. Hickstein. 1998. Retroviral-Mediated Gene Transfer of the Leukocyte Integrin CD18 into Peripheral Blood CD34+ Cells Derived from a Patient with Leukocyte Adhesion Deficiency Type 1. Blood 91: 1520-1526. 9. Kwiatkowski, B.A., L.S. Bastian, T.R. Bauer, Jr., S. Tsai, A.G. Zielinska-Kwiatkowska, and D.D. Hickstein. 1998. The ets Family Member Tel Binds to the Fli-1 Oncoprotein and Inhibits Its Transcriptional Activity. J. Biol. Chem 273: 17525-17530. 10. Kijas, J.M.H., T.R. Bauer, Jr., S. GŠfvert, S. Marklund, G. Trowald-Wigh, A. Johannisson, å. Hedhammar, M. Binns, R.K. Juneja, D.D. Hickstein, and L. Andersson. 1999. A missense mutation in the ( 2-integrin gene (ITGB2) causes canine leukocyte adhesion deficiency. Genomics 61: 101-107. 11. Kwiatkowski, B.A., A.G. Zielinska-Kwiatkowska, T.R. Bauer, Jr., and D.D. Hickstein. 2000. The ETS Family Member Tel Antagonizes the Fli-1 Phenotype in Hematopoietic Cells. Blood Cells Mol. Dis. 26: 84-90. 12. Bauer, Jr. T.R., and D.D. Hickstein. 2000. Gene therapy for leukocyte adhesion deficiency. Curr Op Mol Therapeutics 2(4): 383-388. 13. Creevy, K.E., T.R. Bauer Jr., L.M. Tuschong, L.J. Embree, L. Colenda, K. Cogan, M.F. Starost, M.E. Haskins, D.D. Hickstein. 2003. Canine leukocyte adhesion deficiency colony for investigation of novel hematopoietic therapies. Vet. Immunol. Immunopath. 94:11-22. 14. Creevy, K.E., T.R. Bauer Jr., L.M. Tuschong, L.J. Embree, A.M. Silverstone, J.D. Bacher, C. Romines, J. Garnier, M.L. Thomas 3rd, L. Colenda, and D.D. Hickstein. 2003. Mixed chimeric hematopoietic stem cell transplant reverses the disease phenotype in canine leukocyte adhesion deficiency. Vet. Immunol. Immunopath. 95: 113-121. 15. Bauer, Jr. T.R., Y.C. Gu, K.E. Creevy, L.M. Tuschong, L. Embree, S.M. Holland, R.A. Sokolic, and D.D. Hickstein. 2004. Leukocyte Adhesion Deficiency in Children and Irish Setter Dogs. Ped. Res. 55: 363-367. 16. Bauer, Jr. T.R., K.E. Creevy, Y.C. Gu, L.M. Tuschong, R.E. Donahue, M.E. Metzger, L.J. Embree, T. Burkholder, J.D. Bacher, C. Romines, M.L. Thomas 3rd, L. Colenda, and D.D. Hickstein. 2004. Very low levels of donor CD18+ neutrophils following allogeneic hematopoietic stem cell transplantation reverse the disease phenotype in canine leukocyte adhesion deficiency. Blood 103: 3582-3589. 17. Gu, Y.C., T.R. Bauer Jr., M.R. Ackermann, C.W. Smith, M.E. Kehrli Jr., M.F. Starost, and D.D. Hickstein. 2004. The Genetic Immunodeficiency Disease Leukocyte Adhesion Deficiency in Man, Dog, Cattle, and Mouse. Comparative Med. 54:363-372. 18. Bauer, Jr. T.R., Y.C. Gu, L.M. Tuschong, T. Burkholder, J.D. Bacher , M.F. Starost, R.E. Donahue, R.A. Sokolic, D.D. Hickstein. 2005. Nonmyeloablative hematopoietic stem cell transplantation corrects the disease phenotype in the canine model of leukocyte adhesion deficiency. Exp. Hematol 33:706-712. 19. Vogt G, Chapgier A, Yang K, Chuzhanova N, Feinberg J, Fieschi C, Boisson-Dupuis S, Alcais A, Filipe-Santos O, Bustamante J, de Beaucoudrey L, Al-Mohsen I, Al-Hajjar S, Al-Ghonaium A, Adimi P, Mirsaeidi M, Khalilzadeh S, Rosenzweig S, de la Calle Martin O, Bauer TR, Puck JM, Ochs HD, Furthner D, Engelhorn C, Belohradsky B, Mansouri D, Holland SM, Schreiber RD, Abel L, Cooper DN, Soudais C, Casanova JL. 2005. Gains of glycosylation comprise an unexpectedly large group of pathogenic mutations. Nat Genet. 37:692-700. 20. Sokolic RA, Bauer TR, Gu YC, Hai M, Tuschong LM, Burkholder T, Colenda L, Bacher J, Starost MF, Hickstein DD. 2005. Nonmyeloablative conditioning with busulfan before matched littermate bone marrow transplantation results in reversal of the disease phenotype in canine leukocyte adhesion deficiency. Biol Blood Marrow Transplant. 11:755-763. 21. Gu YC, Bauer TR, Sokolic RA, Hai M, Tuschong LM, Burkholder T, Bacher J, Starost MF, Hickstein DD. 2006. Conversion of the severe to the moderate disease phenotype with donor leukocyte microchimerism in canine leukocyte adhesion deficiency. Bone Marrow Transplant. 37:607-614. 22. Engel ME, Hickstein DD, Bauer Jr TR, Calder C, Manes B, Frangoul H. 2006. Matched unrelated bone marrow transplantation with reduced-intensity conditioning for leukocyte adhesion deficiency. Bone Marrow Transplant. 37:717-718. 23. Burkholder TH, Colenda L, Tuschong LM, Starost MF, Bauer Jr TR, Hickstein DD. 2006. Reproductive capability in dogs with canine leukocyte adhesion deficiency treated with nonmyeloablative conditioning prior to allogeneic hematopoietic stem cell transplantation. Blood. 108:1767-1769. 24. Bauer TR Jr, Hai M, Tuschong LM, Burkholder TH, Gu YC, Sokolic RA, Ferguson C, Dunbar CE, Hickstein DD. 2006. Correction of the disease phenotype in canine leukocyte adhesion deficiency using ex vivo hematopoietic stem cell gene therapy. Blood. 108:3313-3320. 25. Bauer TR Jr, Allen JM, Hai M, Tuschong LM, Khan IF, Olson EM, Adler RL, Burkholder TH, Gu YC, Russell DW, Hickstein DD. 2008. Successful treatment of canine leukocyte adhesion deficiency by foamy virus vectors. Nat Med. 14:93-97. Meeting Presentations Oral Abstract Presentations 1. Bauer, Jr. T.R. and B. Blomberg. A Non-Functional Allele of the Human Immunoglobulin C(6 gene segment. Eastern Student Research Forum, Miami, Florida, March 1989. 2. Bauer, Jr. T.R., A.D. Miller, and D.D. Hickstein, D.D. 1994. Retroviral Vectors and Packaging Cell Lines for Use in Gene Therapy of Leukocyte Adherence Deficiency. American Society of Hematology 36th Annual Meeting. 3. Bauer, Jr. T.R. and D.D. Hickstein, D.D. 1998. Transduction of Hematopoietic CD34+ Cells Derived from a Patient with Leukocyte Adhesion Deficiency Using Retroviral Vectors Containing Leukocyte Integrin Promoters. Keystone Symposia on Molecular and Cellular Biology of Gene Therapy; Gene Therapy Strategies for Hematopoietic Cells. 4. Bauer, Jr., T.R., K.E. Creevy, Y.C. Gu, R.E. Donahue, M.E. Metzger, L.M. Tuschong, L.J. Embree, and D.D. Hickstein. 2003. Very Low-Levels of CD18+ Neutrophils in the Peripheral Blood after Non-Myeloablative Matched Littermate Transplant Corrects the Disease Phenotype in Dogs with Canine Leukocyte Adhesion Deficiency. American Society of Hematology 45th Annual Meeting. 5. Bauer, Jr., T.R., L.M. Tuschong, M. Hai, Y.C. Gu, R.A. Sokolic, T. Burkholder, J.D. Bacher, and D.D. Hickstein. 2005. Reversal of Canine Leukocyte Adhesion Deficiency by Retroviral-Vector Mediated Gene Therapy with Non-Myeloablative Conditioning. American Society of Gene Therapy 8th Annual Meeting. 6. Bauer, Jr., T.R., M. Hai, R.L. Adler, J.M. Allen, L.M. Tuschong, E.M. Olson, D.D. Hickstein, and D.W. Russell. 2005. Therapeutic levels of gene corrected neutrophils following foamy viral vector mediated gene transfer in a canine model of leukocyte adhesion deficiency. American Society of Hematology 47th Annual Meeting. 7. Bauer, Jr., T.R., J.M. Allen, L.M. Tuschong, E.K. Olson, T. Burkholder, M. Hai, Y.C. Gu, R.A. Sokolic, D.D. Hickstein, and D.W. Russell. 2006. Phenotypic correction of four animals with canine leukocyte adhesion deficiency using foamy viral vector-mediated gene therapy. American Society of Gene Therapy 9th Annual Meeting. 8. Bauer, Jr., T.R., J.M. Allen, L.M. Tuschong, E.K. Olson, T.H. Burkholder, M. Hai, Y.C. Gu, D.D. Hickstein, and D.W. Russell. 2007. Successful gene therapy of canine leukocyte adhesion deficiency using foamy viral vectors. American Society of Gene Therapy 10th Annual Meeting. Poster Abstract Presentations (selected) 1. Bauer, Jr. T.R. and D.D. Hickstein 1996. Marking of Normal Human Progenitor Cells by a Novel Reporter Gene, A Truncated Murine CD4. Keystone Symposia on Molecular and Cellular Biology: Gene Therapy for Hematopoietic Stem Cells in Genetic Disease and Cancer. 2. Bauer, Jr. T.R. and Hickstein, D.D. 1997. Transduction of Hematopoietic Cell Lines using Retroviral Vectors containing Leukocyte Integrin Promoters. Keystone Symposia on Molecular and Cellular Biology of Gene Therapy. 3. Bauer, Jr. T.R., B.R. Schwartz, W.C. Liles, H.D. Ochs, and D.D. Hickstein, 1997. Retroviral-mediated gene transfer of human CD18 into CD34+ cells from a patient with Leukocyte Adherence Deficiency: Implications for human gene therapy. American Society of Hematology 39th Annual Meeting. 4. Bauer, Jr. T.R., H.-P. Kiem, J.C. Morris, T.H. Price, H.D. Ochs, S. Heimfeld, S.D. Rowley, R.G. Andrews. A.D. Miller, B. Ramsey, I.D. Bernstein, and D.D. Hickstein. 2000. Retrovirus Mediated Gene Transfer of CD18 into Peripheral Blood Stem Cells in Two Patients with Leukocyte Adhesion Deficiency. American Society of Gene Therapy 3rd Annual Meeting. 5. Bauer, Jr. T.R., K.E. Creevy, Y.C. Gu, L.M. Tuschong, L.J. Embree, and D.D. Hickstein. 2003. Stable Mixed Chimerism Following a Non-Myeloablative Transplant Regimen Corrects the Disease Phenotype in Dogs with the Primary Immunodeficiency Disease Canine Leukocyte Adhesion Deficiency. American Society of Hematology 45th Annual Meeting. 6. Hai, M.H., T.R. Bauer, Y.C. Gu, L.M. Tuschong, R.A. Sokolic, and D.D. Hickstein. 2004. Retroviral-Mediated Gene Transfer of CD18 into Hematopoietic Stem Cells in Dogs with Canine Leukocyte Adhesion Deficiency Reverses the Severe Deficiency Phenotype. American Society of Hematology 47th Annual Meeting. 7. Sokolic, R.A., Y.C. Gu, T. Burkholder, L.M. Tuschong, M. Hai, T.R. Bauer, and D.D. Hickstein. 2004. Non-Myeloablative Conditioning with Busulfan Prior to Allogeneic Hematopoietic Stem Cell Transplant Results in Mixed Chimerism and Reversal of the Phenotype in Canine Leukocyte Adhesion Deficiency with Minimal Regimen Related Toxicity and without Graft-vs.-Host Disease. 8. Gu, Y.C., T.R. Bauer, L.M. Tuschong, R.A. Sokolic, R.E. Donahue, and D.D. Hickstein. 2004. Correction of the Phenotype in Canine Leukocyte Adhesion Deficiency Following Non-Myeloablative, Matched Littermate Transplant. American Society of Hematology 45th Annual Meeting. 9. Bauer, Jr., T.R., M. Hai, R.L. Adler, J.M. Allen, L.M. Tuschong, E.M. Olson, D.D. Hickstein, and D.W. Russell. 2007. Reduced genotoxic risk using foamy viral vectors for the treatment of canine leukocyte adhesion deficiency. American Society of Hematology 49th Annual Meeting. Mentoring 1. Yu-Chen Gu, Ph.D., Post-doctoral Fellow, 10/2002-10/2007 2. Mehreen Hai, Ph.D., Post-doctoral Fellow, 11/2003-11/2007 7