Current international perspectives on hematopoietic stem cell transplantation for inherited metabolic disorders

Pediatric Clinics of North America

Volumn 57, Issue 1, 2010, Pages 123-145

  Boelens, Jaap J ^a   Prasad, Vinod K ^b   Tolar, Jakub ^c   Wynn, Robert F ^d   Peters, Charles ^e,f  

^a UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

^b DUKE UNIVERSITY MEDICAL CENTER   (United States)

^c UNIVERSITY OF MINNESOTA MEDICAL SCHOOL   (United States)

^d ROYAL MANCHESTER CHILDREN S HOSPITAL   (United Kingdom)

^e Sanford Children's Hospital   (United States)

^f UNIVERSITY OF SOUTH DAKOTA   (United States)

Author keywords

Bone marrow transplantation; Hematopoietic stem cell transplantation; Inborn errors of metabolism; Inherited metabolic disorders; Pediatrics; Storage diseases; Umbilical cord blood transplantation

Indexed keywords

BUSULFAN; CYCLOPHOSPHAMIDE; GLUCOSIDASE; GLUCOSYLCERAMIDASE; LEVO IDURONIDASE; N ACETYLGALACTOSAMINE 4 SULFATASE; SPHINGOMYELIN PHOSPHODIESTERASE;

ADRENOLEUKODYSTROPHY; ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; ASPARTYLGLYCOSAMINURIA; CLINICAL TRIAL; CORD BLOOD STEM CELL TRANSPLANTATION; ENZYME REPLACEMENT; FARBER DISEASE; FUCOSIDOSIS; GAUCHER DISEASE; GLOBOID CELL LEUKODYSTROPHY; GLYCOGEN STORAGE DISEASE TYPE 2; GRAFT FAILURE; GRAFT VERSUS HOST REACTION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; HUNTER SYNDROME; HURLER SYNDROME; INBORN ERROR OF METABOLISM; MANNOSIDOSIS; MAROTEAUX LAMY SYNDROME; METACHROMATIC LEUKODYSTROPHY; MUCOLIPIDOSIS TYPE 2; MUCOPOLYSACCHARIDOSIS TYPE 7; NIEMANN PICK DISEASE; PERIPHERAL BLOOD STEM CELL TRANSPLANTATION; REDUCED INTENSITY CONDITIONING; REVIEW; SANFILIPPO SYNDROME; SCHEIE SYNDROME; SURVIVAL RATE; THERAPY EFFECT; TREATMENT INDICATION; TREATMENT OUTCOME; TREATMENT RESPONSE; WOLMAN DISEASE;

BIOMEDICAL RESEARCH; CHILD; GENETIC PREDISPOSITION TO DISEASE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; INTERNATIONAL COOPERATION; METABOLISM, INBORN ERRORS;

EID: 77951072800     PISSN: 00313955     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.pcl.2009.11.004     Document Type: Review

References (116)

1. Fratantoni J.C., Hall C.W., Neufeld E.F. The defect in Hurler and Hunter syndromes. II. Deficiency of specific factors involved in mucopolysaccharide degradation. Proc Natl Acad Sci U S A 1969, 64:360-366.
2. Neufeld E.F. Lysosomal storage diseases. Annu Rev Biochem 1991, 60:257-280.
3. Pfeffer S.R. Targeting of proteins to the lysosome. Curr Top Microbiol Immunol 1991, 170:43-65.
4. Perry V.H., Gordon S. Macrophages and the nervous system. Int Rev Cytol 1991, 125:203-244.
5. Krivit W., Sung J.H., Shapiro E.G., et al. Microglia: the effector cell for reconstitution of the central nervous system following bone marrow transplantation for lysosomal and peroxisomal storage diseases. Cell Transplant 1995, 4:385-392.
6. Giulian D. Ameboid microglia as effectors of inflammation in the central nervous system. J Neurosci Res 1987, 18:155-171.
7. Hickey W.F. Migration of hematogenous cells through the blood-brain barrier and the initiation of CNS inflammation. Brain Pathol 1991, 1:97-105.
8. Kennedy D.W., Abkowitz J.L. Kinetics of central nervous system microglial and macrophage engraftment: analysis using a transgenic bone marrow transplantation model. Blood 1997, 90:986-993.
9. Hobbs J.R., Hugh-Jones K., Barrett A.J., et al. Reversal of clinical features of Hurler's disease and biochemical improvement after treatment by bone-marrow transplantation. Lancet 1981, 2(8249):709-712.
10. Krivit W., Pierpont M.E., Ayaz K., et al. Bone-marrow transplantation in the Maroteaux-Lamy syndrome (mucopolysaccharidosis type VI). N Engl J Med 1984, 311:1606-1611.
11. Aubourg P., Blanche, Jambaque I., et al. Reversal of early neurologic and neuroradiologic manifestations of X-linked adrenoleukodystrophy by bone marrow transplantation. N Engl J Med 1990, 322:1860-1866.
12. Krivit W., Shapiro E., Peters C., et al. Hematopoietic stem-cell transplantation in globoid-cell leukodystrophy. N Engl J Med 1998, 338:1119-1126.
13. Krivit W., Shapiro E., Kennedy W., et al. Treatment of late-infantile metachromatic leukodystrophy by bone marrow transplantation. N Engl J Med 1990, 322:28-32.
14. Vellodi A., Young E., Cooper A., et al. Bone marrow transplantation for mucopolysaccharidosis type I: experience of two British centres. Arch Dis Child 1997, 76:92-99.
15. Peters C., Shapiro E.G., Anderson J., et al. Hurler syndrome. II. Outcome of HLA-genotypically identical sibling and HLA-haploidentical related donor bone marrow transplantation in fifty-four children. The Storage Disease Collaborative Study Group. Blood 1998, 91:2601-2608.
16. Staba S.L., Escolar M.L., Poe M., et al. Cord-blood transplants from unrelated donors in patients with Hurler's syndrome. N Engl J Med 2004, 350:1960-1969.
17. Boelens J.J., Wynn R.F., O'Meara A., et al. Outcomes of hematopoietic stem cell transplantation for Hurler's syndrome in Europe: a risk factor analysis for graft failure. Bone Marrow Transplant 2007, 40:225-233.
18. Boelens J.J., Rocha V., Aldenhoven M., et al. EUROCORD, Inborn Error Working Party of EBMT and Duke University. Risk factor analysis of outcomes after unrelated cord blood transplantation in patients with Hurler syndrome. Biol Blood Marrow Transplant 2009, 15:618-625.
19. Peters C., Charnas L.R., Tan Y., et al. Cerebral X-linked adrenoleukodystrophy: the international hematopoietic cell transplantation experience from 1982 to 1999. Blood 2004, 104:881-888.
20. Beam D., Poe M.D., Provenzale J.M., et al. Outcomes of unrelated umbilical cord blood transplantation for X-linked adrenoleukodystrophy. Biol Blood Marrow Transplant 2007, 13:665-674.
21. Escolar M.L., Poe M.D., Provenzale J.M., et al. Transplantation of umbilical-cord-blood in babies with infantile Krabbe's disease. N Engl J Med 2005, 352:2069-2081.
22. Grigull L., Beilken A., Schrappe M., et al. Transplantation of allogeneic CD34-selected stem cells after fludarabine-based conditioning regimen for children with mucopolysaccharidosis IH (M. Hurler). Bone Marrow Transplant 2005, 35:265-269.
23. Peters C., Balthazor M., Shapiro E.G., et al. Outcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome. Blood 1996, 87:4894-4902.
24. Prasad V.K., Kurtzberg J. Emerging trends in transplantation of inherited metabolic diseases. Bone Marrow Transplant 2008, 41(2):99-108.
25. Prasad V.K., Mendizabal A., Parikh S.H., et al. Unrelated donor umbilical cord blood for inherited metabolic disorders in 159 pediatric patients from a single center: influence of cellular composition of the graft on transplantation outcomes. Blood 2008, 112:2979-2989.
26. Grewal S.S., Wynn R., Abdenur J.E., et al. Safety and efficacy of enzyme replacement therapy in combination with hematopoietic stem cell transplantation in Hurler syndrome. Genet Med 2005, 7:143-146.
27. Cox-Brinkman J., Boelens J.J., Wraith J.E., et al. Hematopoietic cell transplantation (HCT) in combination with enzyme replacement therapy (ERT) in patients with Hurler syndrome. Bone Marrow Transplant 2006, 38:17-21.
28. Tolar J., Grewal S.S., Bjoraker K.J., et al. Combination of enzyme replacement and hematopoietic stem cell transplantation as therapy for Hurler syndrome. Bone Marrow Transplant 2008, 41(6):531-535.
29. Wynn R.F., Mercer J., Page J., et al. Use of enzyme replacement therapy (Laronidase) before hematopoietic stem cell transplantation for mucopolysaccharidosis I: experience in 18 patients. J Pediatr 2009, 154(1):135-139.
30. Peters C. Hematopoietic cell transplantation for storage diseases. Thomas' hematopoietic cell transplantation 2009, 1136-1162. Wiley-Blackwell, Hoboken (NJ). 4th edition. F.R. Appelbaum, S.J. Forman, R.S. Negrin (Eds.).
31. Neufeld E.F., Muenzer J. The Mucopolysaccharidosis. The metabolic and molecular basis of inherited disease 2001, 3421-3452. McGraw-Hill, New York. 8th edition. C. Scriver, A. Beaudet, W. Sly (Eds.).
32. Aldenhoven M., Boelens J.J., de Koning T.J. The clinical outcome of Hurler syndrome after stem cell transplantation. Biol Blood Marrow Transplant 2008, 14(5):485-498.
33. Boelens J.J. Trends in haematopoietic cell transplantation for inborn errors of metabolism. J Inherit Metab Dis 2006, 29(2-3):413-420.
34. Kurtzberg J., Laughlin M., Graham M.L., et al. Placental blood as a source of hematopoietic stem cells for transplantation into unrelated recipients. N Engl J Med 1996, 335(3):157-166.
35. Chen N., Hudson J.E., Walczak P., et al. Human umbilical cord blood progenitors: the potential of these hematopoietic cells to become neural. Stem Cells 2005, 23(10):1560-1570.
36. Kogler G., Sensken S., Airey J.A., et al. A new human somatic stem cell from placental cord blood with intrinsic pluripotent differentiation potential. J Exp Med 2004, 200(2):123-135.
37. Meier C., Middelanis J., Wasielewski B., et al. Spastic paresis after perinatal brain damage in rats is reduced by human cord blood mononuclear cells. Pediatr Res 2006, 9(2):244-249.
38. Church H., Tylee K., Cooper A., et al. Biochemical monitoring after haemopoietic stem cell transplant for Hurler syndrome (MPSIH): implications for functional outcome after transplant in metabolic disease. Bone Marrow Transplant 2007, 39(4):207-210.
39. Martin P.L., Carter S.L., Kernan N.A., et al. Results of the cord blood transplantation study (COBLT): outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with lysosomal and peroxisomal storage diseases. Biol Blood Marrow Transplant 2006, 12(2):184-194.
40. Stein J., Zion G.B., Dror Y., et al. Successful treatment of Wolman disease by unrelated umbilical cord blood transplantation. Eur J Pediatr 2007, 166(7):663-666.
41. Baxter M.A., Wynn R.F., Schyma L., et al. Marrow stromal cells from patients affected by MPS I differentially support haematopoietic progenitor cell development. J Inherit Metab Dis 2005, 28(6):1045-1053.
42. Kakkis E.D., Muenzer J., Tiller G.E., et al. Enzyme-replacement therapy in mucopolysaccharidosis I. N Engl J Med 2001, 344(3):182-188.
43. Wraith J.E. The first 5 years of clinical experience with laronidase enzyme replacement therapy for mucopolysaccharidosis I. Expert Opin Pharmacother 2005, 6(3):489-506.
44. Cimaz R., Vijay S., Haase C., et al. Attenuated type I mucopolysaccharidosis in the differential diagnosis of juvenile idiopathic arthritis: a series of 13 patients with Scheie syndrome. Clin Exp Rheumatol 2006, 24(2):196-202.
45. Wraith J.E., Beck M., Lane R., et al. Enzyme replacement therapy in patients who have mucopolysaccharidosis I and are younger than 5 years: results of a multinational study of recombinant human alpha-L-iduronidase (laronidase). Pediatrics 2007, 120(1):e37-e46.
46. Aldenhoven M., Sakkers R., Boelens J., et al. The musculoskeletal manifestations of lysosomal storage disorders. Ann Rheum Dis 2009, 68(3):1659-1665.
47. Manger B., Mengel E., Schaefer R.M. Rheumatologic aspects of lysosomal storage diseases. Clin Rheumatol 2007, 26(3):335-344.
48. Bergstrom S.K., Quinn J.J., Greenstein R., et al. Long-term follow-up of a patient transplanted for Hunter's disease type IIB: a case report and literature review. Bone Marrow Transplant 1994, 14(4):653-658.
49. Coppa G.V., Gabrielli O., Zampini L., et al. Bone marrow transplantation in Hunter syndrome (mucopolysaccharidosis type II): 2-year follow-up of the first Italian patient and review of the literature. Pediatr Med Chir 1995, 17(3):227-235.
50. Guffon N., Bertrand Y., Forest I., et al. Bone marrow transplantation in children with Hunter syndrome: outcome after 7 to 17 years. J Pediatr 2009, 154(5):733-737.
51. Li P., Thompson J.N., Hug G., et al. Biochemical and molecular analysis in a patient with the severe form of Hunter syndrome after bone marrow transplantation. Am J Med Genet 1996, 64(4):531-535.
52. Peters C., Krivit W. Hematopoietic cell transplantation for mucopolysaccharidosis IIB (Hunter syndrome). Bone Marrow Transplant 2000, 25(10):1097-1099.
53. Vellodi A., Young E., Cooper A., et al. Long-term follow-up following bone marrow transplantation for Hunter disease. J Inherit Metab Dis 1999, 22(5):638-648.
54. Mullen C.A., Thompson J.N., Richard L.A., et al. Unrelated umbilical cord blood transplantation in infancy for mucopolysaccharidosis type IIB (Hunter syndrome) complicated by autoimmune hemolytic anemia. Bone Marrow Transplant 2000, 25(10):1093-1097.
55. Gungor N., Tuncbilek E. Sanfilippo disease type B. A case report and review of the literature on recent advances in bone marrow transplantation. Turk J Pediatr 1995, 37(2):157-163.
56. Vellodi A., Young E., New M., et al. Bone marrow transplantation for Sanfilippo disease type B. J Inherit Metab Dis 1992, 15(6):911-918.
57. Herskhovitz E., Young E., Rainer J., et al. Bone marrow transplantation for Maroteaux-Lamy syndrome (MPS VI): long-term follow-up. J Inherit Metab Dis 1999, 22(1):50-62.
58. Lee V., Li C.K., Shing M.M., et al. Umbilical cord blood transplantation for Maroteaux-Lamy syndrome (mucopolysaccharidosis type VI). Bone Marrow Transplant 2000, 26(4):455-458.
59. Mosser J., Douar A.M., Sarde C.O., et al. Putative X-linked adrenoleukodystrophy gene shares unexpected homology with ABC transporters. Nature 1993, 361:726-730.
60. Boehm C.D., Cutting G.R., Lachtermacher M.B., et al. Accurate DNA-based diagnostic and carrier testing for X-linked adrenoleukodystrophy. Mol Genet Metab 1999, 66:128-136.
61. Moser A.B., Kreiter N., Bezman L., et al. Plasma very long chain fatty acids in 3,000 peroxisome disease patients and 29,000 controls. Ann Neurol 1999, 45:100-110.
62. Bezman L., Moser A.B., Raymond G.V., et al. Adrenoleukodystrophy: incidence, new mutation rate, and results of extended family screening. Ann Neurol 2001, 49:512-517.
63. Aubourg P., Chaussain J.L. Adrenoleukodystrophy: the most frequent genetic cause of Addison's disease. Horm Res 2003, 59(Suppl 1):104-105.
64. Ito M., Blumberg B.M., Mock D.J., et al. Potential environmental and host participants in the early white matter lesion of adreno-leukodystrophy: morphologic evidence for CD8 cytotoxic T cells, cytolysis of oligodendrocytes, and CD1-mediated lipid antigen presentation. J Neuropathol Exp Neurol 2001, 60:1004-1119.
65. Moser H.W. Adrenoleukodystrophy: phenotype, genetics, pathogenesis and therapy. Brain 1997, 120(Pt 8):1485-1508.
66. Hershkovitz E., Narkis G., Shorer Z., et al. Cerebral X-linked adrenoleukodystrophy in a girl with Xq27-Ter deletion. Ann Neurol 2002, 52:234-237.
67. O'Neill G.N., Aoki M., Brown R.H. ABCD1 translation-initiator mutation demonstrates genotype-phenotype correlation for AMN. Neurology 2001, 57:1956-1962.
68. Powers J.M., DeCiero D.P., Cox C., et al. The dorsal root ganglia in adrenomyeloneuropathy: neuronal atrophy and abnormal mitochondria. J Neuropathol Exp Neurol 2001, 60:493-501.
69. Powers J.M., DeCiero D.P., Ito M., et al. Adrenomyeloneuropathy: a neuropathologic review featuring its noninflammatory myelopathy. J Neuropathol Exp Neurol 2000, 59:89-102.
70. Moser H.W., Bergin A., Naidu S., et al. Adrenoleukodystrophy. Endocrinol Metab Clin North Am 1991, 20:297-318.
71. Moser H.W., Naidu S., Kumar A.J., et al. The adrenoleukodystrophies. Crit Rev Neurobiol 1987, 3:29-88.
72. Moser H.S.K., Watkins P., Powers J., et al. X-linked adrenoleukodystrophy. The metabolic and molecular basis of inherited disease 2001, 3257. McGraw-Hill, New York. 8th edition. C. Scriver, B. Beaudet, W. Sly (Eds.).
73. Peters C., Steward C.G. Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines. Bone Marrow Transplant 2003, 31:229-239.
74. Loes D.J., Fatemi A., Melhem E.R., et al. Analysis of MRI patterns aids prediction of progression in X-linked adrenoleukodystrophy. Neurology 2003, 61:369-374.
75. Loes D.J., Hite S., Moser H., et al. Adrenoleukodystrophy: a scoring method for brain MR observations. AJNR Am J Neuroradiol 1994, 15:1761-1766.
76. Shapiro E., Krivit W., Lockman L., et al. Long-term effect of bone-marrow transplantation for childhood-onset cerebral X-linked adrenoleukodystrophy. Lancet 2000, 356:713-718.
77. Tolar J., Orchard P., Bjoraker K., et al. N-acetyl-L-cysteine improves outcome of advanced adrenoleukodystrophy. Bone Marrow Transplant 2007, 39(4):211-215.
78. Moser H.W., Raymond G.V., Koehler W., et al. Evaluation of the preventive effect of glyceryl trioleate-trierucate (" Lorenzo's oil" ) therapy in X-linked adrenoleukodystrophy: results of two concurrent trials. Adv Exp Med Biol 2003, 544:369-387.
79. Wenger D.A., Suzuki K., Suzuki Y., et al. Galactosylceramide lipidosis: globoid-cell leukodystrophy (Krabbe disease). The metabolic and molecular bases of inherited disease 2001, 3669-3694. McGraw-Hill, New York. 8th edition. C.R. Scriver, A.L. Beaudet, W.S. Sly (Eds.).
80. Escolar M.L., Poe M.D., Martin H.R., et al. A staging system for infantile Krabbe disease to predict outcome after unrelated umbilical cord blood transplantation. Pediatrics 2006, 118(3):e879-e889.
81. Duffner P.K., Caggana M., Orsini J.J., et al. Newborn screening for Krabbe disease: the New York State model [comment]. Pediatr Neurol 2009, 40(4):245-252.
82. Stillman A.E., Krivit W., Shapiro E.G., et al. Serial MR after bone marrow transplantation in two patients with metachromatic leukodystrophy. AJNR Am J Neuroradiol 1994, 15:1929-1932.
83. Shapiro E.G., Lipton M.E., Krivit W. White matter dysfunction and its neuropsychological correlates: a longitudinal study of a case of metachromatic leukodystrophy treated with bone marrow transplant. J Clin Exp Neuropsychol 1992, 14:610-624.
84. Pridjian G., Humbert J., Willis J., et al. Pre-symptomatic late-infantile metachromatic leukodystrophy treated with bone marrow transplantation. J Pediatr 1994, 125:755-758.
85. Guffon N., Souillet G., Maire I., et al. Juvenile metachromatic leukodystrophy: neurological outcome two years after bone marrow transplantation. J Inherit Metab Dis 1995, 18:159-161.
86. Shapiro E.G., Lockman L.A., Knopman D., et al. Characteristics of the dementia in late-onset metachromatic leukodystrophy. Neurology 1994, 44:662-665.
87. Navarro C., Fernandez J.M., Dominguez C., et al. Late juvenile metachromatic leukodystrophy treated with bone marrow transplantation: a 4-year follow-up study. Neurology 1996, 46:254-256.
88. Kapaun P., Dittmann R.W., Granitzny B., et al. Slow progression of juvenile metachromatic leukodystrophy 6 years after bone marrow transplantation. J Child Neurol 1999, 14:222-228.
89. Malm G., Ringdén O., Winiarski J., et al. Clinical outcome in four children with metachromatic leukodystrophy treated by bone marrow transplantation. Bone Marrow Transplant 1996, 17(6):1003-1008.
90. Koç O.N., Day J., Nieder M., et al. Allogeneic mesenchymal stem cell infusion for treatment of metachromatic leukodystrophy (MLD) and Hurler syndrome (MPS-IH). Bone Marrow Transplant 2002, 30(4):215-222.
91. Orchard P.J., Blazar B.R., Wagner J., et al. Hematopoietic cell therapy for metabolic disease. J Pediatr 2007, 151:340-346.
92. Tolar J., Petryk A., Khan K., et al. Long-term metabolic, endocrine, and neuropsychological outcome of hematopoietic cell transplantation for Wolman disease. Bone Marrow Transplant 2009, 43:21-27.
93. Grewal S.S., Shapiro E.G., Krivit W., et al. Effective treatment of alpha-mannosidosis by allogeneic hematopoietic stem cell transplantation. J Pediatr 2004, 144:569-573.
94. Yamada Y., Kato K., Sukegawa K., et al. Treatment of MPS VII (Sly disease) by allogeneic BMT in a female with homozygous A619V mutation. Bone Marrow Transplant 1998, 21:629-634.
95. Klein K.A., Krivit W., Whitley C.B., et al. Poor cognitive outcome of eleven children with Sanfilippo syndrome after bone marrow transplantation and successful engraftment. Bone Marrow Transplant 1995, 15:S176-S181.
96. Grewal S., Shapiro E., Braunlin E., et al. Continued neurocognitive development and prevention of cardiopulmonary complications after successful BMT for I-cell disease: a long-term follow-up report. Bone Marrow Transplant 2003, 32:957-960.
97. Bayever E., Kamani N., Ferreira P., et al. Bone marrow transplantation for Niemann-Pick type IA disease. J Inherit Metab Dis 1992, 15:919-928.
98. Lake B.D., Steward C.G., Oakhill A., et al. Bone marrow transplantation in late infantile Batten disease and juvenile Batten disease. Neuropediatrics 1997, 28:80-81.
99. Jacobs J.F., Willemsen M.A., Groot-Loonen J.J., et al. Allogeneic BMT followed by substrate reduction therapy in a child with subacute Tay-Sachs disease. Bone Marrow Transplant 2005, 36:925-926.
100. Ringdén O., Groth C.G., Erikson A., et al. Longterm follow-up of the first successful bone marrow transplantation in Gaucher disease. Transplantation 1988, 46:66-70.
101. Hobbs J.R., Jones K.H., Shaw P.J., et al. Beneficial effect of pre-transplant splenectomy on displacement bone marrow transplantation for Gaucher's syndrome. Lancet 1987, 1:1111-1115.
102. Zimran A., Bembi B., Pastores G.M. Enzyme replacement therapy for type I Gaucher disease. Gaucher disease 2007, 341-354. Taylor & Francis, London. A.H. Futerman, A. Zimran (Eds.).
103. Erikson A., Groth C.G., Månsson J.E., et al. Clinical and biochemical outcome of marrow transplantation for Gaucher disease of the Norrbottnian type. Acta Paediatr Scand 1990, 79:680-685.
104. Ringdén O., Groth C.G., Erikson A., et al. Ten years' experience of bone marrow transplantation for Gaucher disease. Transplantation 1995, 59:864-870.
105. Erikson A., Forsberg H., Nilsson M., et al. Ten years' experience of enzyme infusion therapy of Norrbottnian (type 3) Gaucher disease. Acta Paediatr 2006, 95:312-317.
106. Porteus M.H., Connelly J.P., Pruett S.M. A look to future directions in gene therapy research for monogenic diseases. PLoS Genet 2006, 2. e133.
107. Biffi A., De Palma M., Quattrini A., et al. Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells. J Clin Invest 2004, 113:1118-1129.
108. Chung S., Ma X., Liu Y., et al. Effect of neonatal administration of a retroviral vector expressing alpha-L-iduronidase upon lysosomal storage in brain and other organs in mucopolysaccharidosis I mice. Mol Genet Metab 2007, 90:181-192.
109. Kobayashi H., Carbonaro D., Pepper K., et al. Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector. Mol Ther 2005, 11:776-789.
110. Liu Y., Xu L., Hennig A.K., et al. Liver-directed neonatal gene therapy prevents cardiac, bone, ear, and eye disease in mucopolysaccharidosis I mice. Mol Ther 2005, 11:35-47.
111. Zheng Y., Rozengurt N., Ryazantsev S., et al. Treatment of the mouse model of mucopolysaccharidosis I with retrovirally transduced bone marrow. Mol Genet Metab 2003, 79:233-244.
112. Hacein-Bey-Abina S., Von Kalle C., Schmidt M., et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 2003, 302:415-419.
113. Pardridge W.M. Molecular biology of the blood-brain barrier. Mol Biotechnol 2005, 30:57-70.
114. Phinney D.G., Prockop D.J. Concise review: mesenchymal stem/multipotent stromal cells: the state of transdifferentiation and modes of tissue repair-current views. Stem Cells 2007, 25:2896-2902.
115. Aggarwal S., Pittenger M.F. Human mesenchymal stem cells modulate allogeneic immune cell responses. Blood 2005, 105:1815-1822.
116. Le Blanc K., Rasmusson I., Sundberg B., et al. Treatment of severe acute graft-versus-host disease with third party haploidentical mesenchymal stem cells. Lancet 2004, 363:1439-1441.