Hematopoietic stem cell transplantation, stem cells, and gene therapy

Gaucher Disease

Volumn , Issue , 2006, Pages 423-437

  Peters, Charles ^a   Krivit, William ^b  

^a UNIVERSITY OF MISSOURI KANSAS CITY   (United States)

^b UNIVERSITY OF MINNESOTA MEDICAL SCHOOL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

EID: 48949121014     PISSN: None     EISSN: None     Source Type: Book    
DOI: 10.1201/9781420005509     Document Type: Chapter

References (76)

1. Bach, F.H. et al., Bone-marrow transplantation in a patient with the Wiskott-Aldrich syndrome. Lancet. 2, 1364, 1968.
2. Gatti, R.A. et al., Immunological reconstitution of sex-linked immunological deficiency. Lancet. 2, 1366, 1968.
3. Good, R.A. et al., Successful marrow transplantation for correction of immunological deficit in lymphopenic agammaglobulinemia and treatment of immunologically induced pancytopenia. Exp. Hematol., 19, 4, 1969.
4. De Konig, J. et al., Transplantation of bone-marrow cells and fetal thymus in an infant with lymphopenic immunological deficiency. Lancet. 1, 1223, 1969.
5. Ringdén, O. et al., Long-term follow-up of the first successful bone marrow transplantation in Gaucher disease. Transplantation. 46, 66, 1988.
6. Bodmer, J.G. et al., Nomenclature for factors of the HLA system. Tissue Antigens. 57, 236, 2001.
7. Mickelson, E. and Petersdorf, E.W., Histocompatibility, in Thomas’ Hematopoietic Cell Transplantation, Blume, K.G., Forman, S.J., and Appelbaum, F.R., Eds., 3rd ed., Blackwell Publishing, Malden, MA, 2004, chap. 4.
8. Bensinger, W.I. and Spielberger, R., Preparative regimens and modification of regimen- related toxicities, in Thomas’ Hematopoietic Cell Transplantation, Blume, K.G., Forman, S.J., and Appelbaum, F.R., Eds., 3rd ed., Blackwell Publishing, Malden, MA, 2004, chap. 13.
9. Sullivan, K.M., Graft-vs.-host disease, in Thomas’ Hematopoietic Cell Transplantation, Blume, K.G., Forman, S.J., and Appelbaum, F.R., Eds., 3rd ed., Blackwell Publishing, Malden, MA, 2004, chap. 50.
10. Broxmeyer, H.E. and Smith, F.O., Cord blood hematopoietic cell transplantation, in Thomas’ Hematopoietic Cell Transplantation, Blume, K.G., Forman, S.J., and Appelbaum, F.R., Eds., 3rd ed., Blackwell Publishing, Malden, MA, 2004, chap. 43.
11. Barker, J.N. and Wagner, J.E., Umbilical cord blood transplantation: current practice and future innovations. Crit. Rev. Oncol. Hematol., 48, 35, 2003.
12. Barker, J.N. et al., Transplantation of 2 partially HLA-matched umbilical cord blood units to enhance engraftment in adults with hematologic malignancy. Blood. 105, 1343, 2005.
13. Fratantoni, J., Hall, C., and Neufeld, E., The defect in Hurler and Hunter syndromes. II. Deficiency of specific factors involved in mucopolysaccharide degradation, Proc. Natl. Acad. Sci. U.S.A., 64, 360, 1969.
14. Di Ferrante, N., Nichols, B., Donnelly, P., Neri, G., Hrgovcic, R., Berglund, R., Induced degradation of glycosaminoglycans in Hurler’s and Hunter’s syndromes by plasma infusion, Proc. Natl. Acad. Sci. U.S.A., 68, 303, 1971.
15. Knudson, A., Di Ferrante, N., and Curtis, J., Effect of leukocyte transfusion in a child with type II mucopolysaccharidosis, Proc. Natl. Acad. Sci. U.S.A., 68, 1738, 1971.
16. Hobbs, J. et al., Reversal of clinical features of Hurler’s disease and biochemical improvement after treatment by bone-marrow transplantation, Lancet. 2, 709, 1981.
17. Rappeport, J.M. and Ginns, E.I., Bone-marrow transplantation in severe Gaucher disease. N. Engl. J. Med., 311, 84, 1984.
18. Hobbs, J.R., Experience with bone marrow transplantation for inborn errors of metabolism. Enzyme. 38, 194, 1987.
19. Hobbs, J.R. et al., Beneficial effect of pre-transplant splenectomy on displacement bone marrow transplantation for Gaucher’s disease. Lancet. 1, 1111, 1987.
20. August, C. et al., Bone marrow transplantation (BMT) in Gaucher’s disease. Pediatr. Res., 18, 236a, 1984.
21. Young, E. et al., Plasma chitotriosidase activity in Gaucher disease patients who have been treated either by bone marrow transplantation or by enzyme replacement therapy with alglucerase, J. Inherit. Metab. Dis., 20, 595, 1997.
22. Yabe, H. et al., Secondary G-CSF mobilized blood stem cell transplantation without preconditioning in a patient with Gaucher disease: report of a new approach which resulted in complete reversal of severe skeletal involvement, Tokai J. Exp. Clin. Med., 30, 77, 2005.
23. Jones, S. et al., DBMT for Gaucher’s disease, in Correction of Certain Genetic Diseases by Transplantation, Hobbs, J.R., Ed., Headstart Printing Co., London, 1989, 23.
24. Erikson, A., Gaucher disease - Norrbottnian type (III). Neuropaediatric and neurobiological aspects of clinical patterns and treatment, Acta Paediatr. Scand. Suppl., 326, 1, 1986.
25. Erikson, A. et al., Clinical and biochemical outcome of marrow transplantation for Gaucher disease of the Norrbottnian type. Acta Paediatr. Scand., 79, 680, 1990.
26. Ringdén, O. et al., Long-term follow-up of the first successful bone marrow transplantation in Gaucher disease. Transplantation. 46, 66, 1988.
27. Lonnqvist, B. et al., Cytomegalovirus infection associated with and preceding chronic graft-versus-host disease. Transplantation. 38, 465, 1984.
28. Tsai, P. et al. Allogeneic bone marrow transplantation in severe Gaucher disease. Pediatr. Res., 31, 503, 1992.
29. Svennerholm, L. et al., Norrbottnian type of Gaucher disease - Clinical, biochemical and molecular biology aspects: successful treatment with bone marrow transplantation. Dev. Neurosci. 13, 345, 1991.
30. Ringdén, O. et al., Ten years’ experience of bone marrow transplantation for Gaucher disease. Transplantation. 59, 864, 1995.
31. Chan, K.W., et al., Bone marrow transplantation in Gaucher’s disease: effect of mixed chimeric state. Bone Marrow Transplant., 14, 327, 1994.
32. Yen, C.C. et al., Allogenic bone marrow transplantation for Gaucher disease - a case report, Zhonghua Yi Xue Za Zhi (Taipei), 59, 372, 1997.
33. Erikson, A., Astrom, M., and Mansson, J.E., Enzyme infusion therapy of the Norrbottnian (type 3) Gaucher disease, Neuropediatrics. 26, 203, 1995.
34. Kalervo Vaananen, H., Mesenchymal stem cells, Ann. Med., 37, 469, 2005.
35. Price, M.J. et al., Intravenous mesenchymal stem cell therapy early after reperfused acute myocardial infarction improves left ventricular function and alters electrophysiologic properties, Int. J. Cardiol., Epub ahead of print, 2005.
36. Koc, O.N. et al., Allogeneic mesenchymal stem cell infusion for treatment of metachromatic leukodystrophy (MLD) and Hurler syndrome (MPS-IH), Bone Marrow Transplant., 30, 215, 2002.
37. Lindvall, O. and Kokaia, Z., Stem cell therapy for human brain disorders. Kidney Int., 68, 1937, 2005.
38. Leker, R.R. and McKay, R.D., Using endogenous neural stem cells to enhance recovery from ischemic brain injury. Curr. Neurovasc. Res., 1, 421, 2004.
39. Mueller, D. et al., Transplanted human embryonic germ cell-derived neural stem cells replace neurons and oligodendrocytes in the forebrain of neonatal mice with excitotoxic brain damage, J. Neurosci. Res., Epub ahead of print, 2005.
40. Ortiz-Conzalez, X.R. et al., Neural induction of adult bone marrow and umbilical cord stem cells, Curr. Neurovasc. Res., 1, 207, 2004.
41. Becker, P.S., The current status of gene therapy in autologous transplantation, Acta Haematol., 114, 188, 2005.
42. Weinthal, J. et al., Expression of human glucocerebrosidase following retroviral vector-mediated transduction of murine hematopoietic stem cells, Bone Marrow Transplant., 8, 403, 1991.
43. Nolta, J.A. et al., Retroviral-mediated transfer of human glucocerebrosidase gene into cultured Gaucher bone marrow, J. Clin. Invest., 90, 342, 1992.
44. Ohashi, T. et al., Efficient transfer and sustained high expression of the human glucocerebrosidase gene in mice and their functional macrophages following transplantation of bone marrow transduced by a retroviral vector, Proc. Natl. Acad. Sci. U.S.A., 89, 11332, 1992.
45. Karlsson, S., Correll, P.H., and Xu, L., Gene transfer and bone marrow transplantation with special reference to Gaucher’s disease, Bone Marrow Transplant., 11, Suppl. 1, 124, 1993.
46. Krall, W.J. et al., Cells expressing human glucocerebrosidase from a retroviral vector repopulate macrophages and central nervous system microglia after murine bone marrow transplantation, Blood. 83, 2737, 1994.
47. Kiem, H.P. et al., Gene therapy and bone marrow transplantation, Curr. Opin. Oncol., 7, 107, 1995.
48. Wells, S. et al., The presence of an autologous marrow stromal cell layer increases glucocerebrosidase gene transduction of long-term culture initiating cells (LTCICs) from the bone marrow of a patient with Gaucher disease, Gene Ther., 2, 512, 1995.
49. Hallek, M. and Wendtner, C.M., Recombinant adeno-associated virus (rAAV) vectors for somatic gene therapy: recent advances and potential clinical applications, Cytokines Mol. Ther., 2, 69, 1996.
50. Havenga, M. et al., Development of safe and efficient retroviral vectors for Gaucher disease, Gene Ther., 4, 1393, 1997.
51. Barranger, J.A. et al., Gaucher’s disease: studies of gene transfer to haematopoietic cells, Baillieres Clin. Haematol., 10, 765, 1997.
52. Takiyama, N. et al., Comparison of methods for retroviral mediated transfer of glucocerebrosidase gene to CD34+ hematopoietic progenitor cells, Eur. J. Haematol., 61, 1, 1998.
53. Robbins, P.D. and Ghivizzani, S.C., Viral vectors for gene therapy, Pharmacol. Ther., 80, 35, 1998.
54. Havenga, M.J. et al., Methotrexate selectable retroviral vectors for Gaucher disease, Gene Ther., 5, 1379, 1998.
55. Dunbar, C.E. et al., Retroviral transfer of the glucocerebrosidase gene into CD34+ cells from patients with Gaucher disease: in vivo detection of transduced cells without myeloablation, Hum. Gene Ther., 9, 2629, 1998.
56. Havenga, M. et al., In vivo methotrexate selection of murine hemopoietic cells transduced with a retroviral vector for Gaucher disease, Gene Ther., 6, 1661, 1999.
57. Shimizu, T. et al., A simple and efficient purification of transduced cells by using green fluorescent protein gene as a selection marker, Acta Paediatr. Jpn., 40, 586, 1998.
58. McIvor, R.S., Gene therapy of genetic diseases and cancer, Pediatr Transplant., 3, Suppl. 1, 116, 1999.
59. Barranger, J.A., Rice, E.O., and Swaney, W.P., Gene transfer approaches to the lysosomal storage disorders, Neurochem. Res., 24, 601, 1999.
60. Barranger, J.A. and Novelli, E.A., Gene therapy for lysosomal storage disorders, Expert Opin. Biol. Ther., 1, 857, 2001.
61. Cabrera-Salazar, M.A., Novelli, E., and Barranger, J.A., Gene therapy for the lysosomal storage disorders, Curr. Opin. Mol. Ther., 4, 349, 2002.
62. De Fost, M., Aerts, J.M., and Hollak, C.E., Gaucher disease: from fundamental research to effective therapeutic interventions, Neth. J. Med., 61, 3, 2003.
63. Grabowski, G.A., Perspectives on gene therapy for lysosomal storage diseases that affect hematopoiesis, Curr. Hematol. Rep., 2, 356, 2003.
64. Marshall, J. et al., Demonstration of the feasibility of in vivo gene therapy for Gaucher disease using a chemically induced mouse model, Mol. Ther., 6, 179, 2002.
65. Diaz-Font, A. et al., Unsuccessful chimeraplast strategy for the correction of a mutation causing Gaucher disease, Blood Cells Mol. Dis., 31, 183, 2003.
66. Campbell, T.N. and Choy, F.Y., Knockdown of chimeric glucocerebrosidase by green fluorescent protein-directed small interfering RNA, Genet. Mol. Res., 3, 282, 2004.
67. Kim, E.Y. et al., Expression and secretion of human glucocerebrosidase mediated by recombinant lentivirus vectors in vitro and in vivo: implications for gene therapy of Gaucher disease, Biochem. Biophys. Res. Commun., 318, 381, 2004.
68. Hong, Y.B. et al., Feasibility of gene therapy in Gaucher disease using an adenoassociated virus vector, J. Hum. Genet., 49, 536, 2004.
69. Erikson, A., Remaining problems in the management of patients with Gaucher disease, J. Inherit. Metab. Dis., 24, Suppl. 2, 122, 2001.
70. Peters, C. et al., Cerebral X-linked adrenoleukodystrophy: the international hematopoietic cell transplantation experience from 1982 to 1999, Blood. 104, 881, 2004.
71. Jmoudiak, M. and Futerman, A.H., Gaucher disease: pathological mechanisms and modern management, Br. J. Haematol., 129, 178, 2005.
72. Germain, D.P., Gaucher’s disease: a paradigm for interventional genetics, Clin. Genet. 65, 77, 2004.
73. Grabowski, G.A., Recent clinical progress in Gaucher disease, Curr. Opin. Pediatr., 17, 519, 2005.
74. Elstein, D. et al., Gaucher disease: pediatric concerns, Paediatr. Drugs. 4, 417, 2002.
75. Schiffmann, R. and Brady, R.O., New prospects for the treatment of lysosomal storage diseases, Drugs. 62, 733, 2002.
76. Balicki, D. and Beutler, E., Gene therapy of human disease, Medicine (Baltimore), 81, 69, 2002.