Bone marrow transplantation combined with gene therapy to induce antigen-specific tolerance and ameliorate EAE

Molecular Therapy

Volumn 13, Issue 1, 2006, Pages 42-48

  Xu, Biying ^a   Haviernik, Peter ^b   Wolfraim, Lawrence A ^c   Bunting, Kevin D ^b,d   Scott, David W ^a  

^a UNIVERSITY OF MARYLAND SCHOOL OF MEDICINE   (United States)

^b CASE WESTERN RESERVE UNIVERSITY   (United States)

^c MacroGenics Inc   (United States)

^d CASE WESTERN RESERVE UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

Autoimmune disease; Bone marrow transplantation; Gene therapy

Indexed keywords

FLUOROURACIL; GREEN FLUORESCENT PROTEIN; PEPTIDE DERIVATIVE; PERTUSSIS TOXIN; PROTEOLIPID PROTEIN; RETROVIRUS VECTOR;

ALLERGIC ENCEPHALOMYELITIS; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTIGEN SPECIFICITY; ARTICLE; BONE MARROW CELL; BONE MARROW TRANSPLANTATION; CELL LINEAGE; CENTRAL NERVOUS SYSTEM; CONTROLLED STUDY; FEMALE; GENE THERAPY; GENETIC TRANSDUCTION; HEMATOPOIETIC CELL; IMMUNIZATION; IMMUNOLOGICAL TOLERANCE; IRRADIATION; LYMPHOCYTE PROLIFERATION; MOUSE; NEUROPROTECTION; NONHUMAN; NONMYELOABLATIVE STEM CELL TRANSPLANTATION; PERIPHERAL CIRCULATION; PROTEIN EXPRESSION; PROVOCATION TEST; T LYMPHOCYTE; TRANSGENE; VIRAL GENE DELIVERY SYSTEM;

ANIMALS; ANTIGENS, CD4; BONE MARROW CELLS; BONE MARROW TRANSPLANTATION; CELL PROLIFERATION; COMBINED MODALITY THERAPY; ENCEPHALOMYELITIS, AUTOIMMUNE, EXPERIMENTAL; FEMALE; GENE THERAPY; GENETIC VECTORS; GREEN FLUORESCENT PROTEINS; HUMANS; IMMUNE TOLERANCE; IMMUNIZATION; L-SELECTIN; MICE; MYELIN PROTEOLIPID PROTEIN; PEPTIDE FRAGMENTS; RECEPTORS, INTERLEUKIN-2; T-LYMPHOCYTES;

ANIMALIA;

EID: 28844477590     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ymthe.2005.09.002     Document Type: Article

References (27)

1. G. Elkin T.B. Prigozhina S. Slavin Prevention of diabetes in nonobese diabetic mice by nonmyeloablative allogeneic bone marrow transplantation Exp. Hematol. 32 2004 579-584
2. C. Tian J. Bagley N. Cretin N. Seth K.W. Wucherpfennig J. Iacomini Prevention of type 1 diabetes by gene therapy J. Clin. Invest. 114 2004 969-978
3. D.W. van Bekkum Stem cell transplantation in experimental models of autoimmune disease J. Clin. Immunol. 20 2000 10-16
4. O.Y. Jones et al. Non-myeloablative bone marrow transplantation of BXSB lupus using fully matched allogeneic donor cells from green fluorescent protein transgenic mice J. Immunol. 172 2004 5415-5419
5. A. Fassas et al. Hematopoietic stem cell transplantation for multiple sclerosis: A retrospective multicenter study J. Neurol. 249 2002 1088-1097
6. + selected autologous peripheral blood stem cell transplantation for multiple sclerosis: Report of toxicity and treatment results at one year of follow-up in 15 patients Haematologica 88 2003 306-314
7. J. Couzin Immunology: Gently soothing a savage immune system Science 296 2002 456-458
8. P.A. Muraro R. Martin Immunological questions on hematopoietic stem cell transplantation for multiple sclerosis Bone Marrow Transplant. 32 2003 S41-S44
9. B.Y. Xu D.W. Scott A novel retroviral gene therapy approach to inhibit specific antibody production and suppress experimental autoimmune encephalomyelitis induced by MOG and MBP Clin. Immunol. 111 2004 47-52
10. M. Melo et al. Gene transfer of immunoglobulin-fusion proteins into B cells prevents and treats autoimmune diseases J. Immunol. 168 2002 4788-4795
11. M.A. Domenick S.T. Ildstad Impact of bone marrow transplantation on type I diabetes World J. Surg. 25 2001 474-480
12. D. Karussis S. Slavin Hematopoietic stem cell transplantation in multiple sclerosis: Experimental evidence to rethink the procedures J. Neurol. Sci. 223 2004 59-64
13. M. van Gelder D.W. van Bekkum Effective treatment of relapsing experimental autoimmune encephalomyelitis with pseudoautologous bone marrow transplantation Bone Marrow Transplant. 18 1996 1029-1034
14. E. Fuchs P. Matzinger B cells turn off virgin but not memory T cells Science 258 1992 1156-1159
15. K. Gilbert W. Weigle Tolerogenicity of resting and activated B cells J. Exp. Med. 179 1994 249-258
16. E. Zambidis A. Kurup D.W. Scott Genetically-transferred central and peripheral immune tolerance via retroviral-mediated expression of immunogenic epitopes in hematopoietic progenitors or peripheral B lymphocytes Mol. Med. 3 1997 212-224
17. R.K. Agarwal Y. Kang E. Zambidis D.W. Scott C.C. Chan R.R. Caspi Retroviral gene transfer of immunoglobulin-antigen fusion protein protects from autoimmune disease J. Clin. Invest. 106 2000 245-252
18. L. Song et al. B-cell delivery of GAD-Ig fusion construct induces specific tolerance and prevents diabetes in NOD mice: Role of CD4 regulatory cells and TGF-β Gene Ther. 11 2004 1487-1496
19. T.-C. Lei D.W. Scott Induction of tolerance to fVIII inhibitors by gene therapy with immunodominant A2 and C2 domains presented by B-cells as Ig fusion proteins Blood 105 2005 4865-4870
20. M. El-Amine M. Melo Y. Kang H. Nguyen J. Qian D.W. Scott Mechanisms of tolerance induction by a gene transferred peptide-IgG fusion protein expressed in B-lineage cells J. Immunol. 165 2000 5631-5636
21. B.L. McRae C.L. Vanderlugt M.C. Dal Canto S.D. Miller Functional evidence for epitope spreading in the relapsing pathology of experimental autoimmune encephalomyelitis J. Exp. Med. 182 1995 75-85
22. T.R. Peters et al. Retrovirus mediated gene transfer of the self antigen MBP into the bone marrow of mice alters resistance to experimental autoimmune encephalomyelitis J. Neuroimmunol. 103 2000 51-62
23. B. Ally et al. Prevention of autoimmune disease by retroviral-mediated gene therapy J. Immunol. 155 1995 5404-5408
24. M. Sykes D.H. Sachs A.W. Nienhuis D.A. Pearson A.D. Moulton D.M. Bodine Specific prolongation of skin graft survival following retroviral transduction of bone marrow with an allogeneic major histocompatibility complex gene Transplantation 55 1993 197-202
25. J. Goolsby et al. Hematopoietic progenitors express neural genes Proc. Natl. Acad. Sci. USA 100 2003 14926-14931
26. S. Bonilla P. Alarcon R. Villaverde P. Aparicio A. Silva S. Martinez Haematopoietic progenitor cells from adult bone marrow differentiate into cells that express oligodendroglial antigens in the neonatal mouse brain Eur. J. Neurosci. 15 2002 575-582
27. K.D. Bunting M.Y. Sangster J.N. Ihle B.P. Sorrentino Restoration of lymphocyte function in Janus kinase 3-deficient mice by retroviral-mediated gene transfer Nat. Med. 4 1998 58-64