메뉴 건너뛰기




Volumn 6, Issue 1, 1999, Pages 107-113

Autologous transplantation of retrovirally transduced bone marrow or neonatal blood cells into cats can lead to long-term engraftment in the absence of myeloablation

Author keywords

Animal models; Hematopoeitic stem cell gene therapy; Mucopolysaccaridosis type VI; Retrovirus mediated gene transfer

Indexed keywords

COMPLEMENTARY DNA; MYELOABLATIVE AGENT; N ACETYLGALACTOSAMINE 4 SULFATASE;

EID: 0032922212     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.gt.3300797     Document Type: Article
Times cited : (33)

References (28)
  • 1
    • 0000820862 scopus 로고
    • The mucopolysaccharidoses
    • Scriver CR, Beaudet AL, Sly WS, Valle D (eds). McGraw-Hill: New York
    • Neufeld EF, Muenzer J. The mucopolysaccharidoses. In: Scriver CR, Beaudet AL, Sly WS, Valle D (eds). The Metabolic and Molecular Bases of Inherited Disease. McGraw-Hill: New York, 1995, pp 2465-2494.
    • (1995) The Metabolic and Molecular Bases of Inherited Disease , pp. 2465-2494
    • Neufeld, E.F.1    Muenzer, J.2
  • 2
    • 0017758232 scopus 로고
    • Mucopolysaccharidosis in a cat with arylsulfatase B deficiency: A model of Maroteaux-Lamy syndrome
    • Jezyk P, Haskins ME, Patterson DF. Mucopolysaccharidosis in a cat with arylsulfatase B deficiency: a model of Maroteaux-Lamy syndrome. Science 1977; 198: 834-836.
    • (1977) Science , vol.198 , pp. 834-836
    • Jezyk, P.1    Haskins, M.E.2    Patterson, D.F.3
  • 3
    • 6544284042 scopus 로고    scopus 로고
    • A clinical vignette. Mucopolysaccharidosis VI (Maroteaux-Lamy syndrome) in a miniature pinscher
    • Neer TM et al. A clinical vignette. Mucopolysaccharidosis VI (Maroteaux-Lamy syndrome) in a miniature pinscher. J Vet Int Med 1997; 6: 429-131.
    • (1997) J Vet Int Med , vol.6 , pp. 429-1131
    • Neer, T.M.1
  • 4
    • 0027408269 scopus 로고
    • Arylsulfatase B-deficient mucopolysaccharidosis in rats
    • Yoshida M et al. Arylsulfatase B-deficient mucopolysaccharidosis in rats. J Clin Invest 1993; 91: 1099-1104.
    • (1993) J Clin Invest , vol.91 , pp. 1099-1104
    • Yoshida, M.1
  • 5
    • 0021683336 scopus 로고
    • Bone-marrow transplantation in the Maroteaux-Lamy syndrome (mucopolysaccharidosis type VI)
    • Krivit W et al. Bone-marrow transplantation in the Maroteaux-Lamy syndrome (mucopolysaccharidosis type VI). New Engl J Med 1984; 311: 1606-1611.
    • (1984) New Engl J Med , vol.311 , pp. 1606-1611
    • Krivit, W.1
  • 6
    • 0022478629 scopus 로고
    • Bone marrow transplantation in Maroteaux-Lamy syndrome (mps type 6): Status 40 months after BMT
    • McGovern MM et al. Bone marrow transplantation in Maroteaux-Lamy syndrome (mps type 6): status 40 months after BMT. Birth Defects 1986; 22: 41-53.
    • (1986) Birth Defects , vol.22 , pp. 41-53
    • McGovern, M.M.1
  • 7
    • 0031029536 scopus 로고    scopus 로고
    • Enzyme replacement therapy from birth in a feline model of mucopolysaccharidosis type VI
    • Crawley AC et al. Enzyme replacement therapy from birth in a feline model of mucopolysaccharidosis type VI. J Clin Invest 1997; 99: 651-662.
    • (1997) J Clin Invest , vol.99 , pp. 651-662
    • Crawley, A.C.1
  • 8
    • 0021679851 scopus 로고
    • Correction of feline arylsulfatase B deficiency (mucopolysaccharidosis VI) by bone marrow transplantation
    • Gasper PW et al. Correction of feline arylsulfatase B deficiency (mucopolysaccharidosis VI) by bone marrow transplantation. Nature 1984; 312: 467-469.
    • (1984) Nature , vol.312 , pp. 467-469
    • Gasper, P.W.1
  • 9
    • 0022535619 scopus 로고
    • Bone marrow transplantation in the feline model of arylsulfatase B deficiency
    • Wenger DA et al. Bone marrow transplantation in the feline model of arylsulfatase B deficiency. Birth Defects 1986; 22: 177-186.
    • (1986) Birth Defects , vol.22 , pp. 177-186
    • Wenger, D.A.1
  • 10
    • 0027285264 scopus 로고
    • Characterization of osteopenia in feline mucopolysaccharidosis VI and evaluation of bone marrow transplantation therapy
    • Norrdin RW, Moffat KS, Thrall MA, Gasper PW. Characterization of osteopenia in feline mucopolysaccharidosis VI and evaluation of bone marrow transplantation therapy. Bone 1993; 14: 361-367.
    • (1993) Bone , vol.14 , pp. 361-367
    • Norrdin, R.W.1    Moffat, K.S.2    Thrall, M.A.3    Gasper, P.W.4
  • 11
    • 0030961365 scopus 로고    scopus 로고
    • Bone marrow transplantation in newborn rats with mucopolysaccharidosis type VI
    • Simonaro CM et al. Bone marrow transplantation in newborn rats with mucopolysaccharidosis type VI. Transplantation 1997; 63: 1386-1393.
    • (1997) Transplantation , vol.63 , pp. 1386-1393
    • Simonaro, C.M.1
  • 12
    • 0029634283 scopus 로고
    • Allogeneic bone marrow transplantation for lysosomal storage diseases
    • Hoogerbrugge PM et al. Allogeneic bone marrow transplantation for lysosomal storage diseases. Lancet 1995; 345: 1398-1402.
    • (1995) Lancet , vol.345 , pp. 1398-1402
    • Hoogerbrugge, P.M.1
  • 13
    • 0026584294 scopus 로고
    • Proliferation of totipotent hematopoietic stem cells in vitro with retention of long-term competitive in vivo reconstiuting ability
    • Fraser CC, Szilvassy SJ, Eaves CJ, Humphries RK. Proliferation of totipotent hematopoietic stem cells in vitro with retention of long-term competitive in vivo reconstiuting ability. Cell Biol 1992; 89: 1968-1972.
    • (1992) Cell Biol , vol.89 , pp. 1968-1972
    • Fraser, C.C.1    Szilvassy, S.J.2    Eaves, C.J.3    Humphries, R.K.4
  • 14
    • 0028145310 scopus 로고
    • Retroviral vector design for long-term expression in murine hematopoietic cells in vivo
    • Correll PH, Colilla S, Karlsson SA. Retroviral vector design for long-term expression in murine hematopoietic cells in vivo. Blood 1994; 84: 1812-1822.
    • (1994) Blood , vol.84 , pp. 1812-1822
    • Correll, P.H.1    Colilla, S.2    Karlsson, S.A.3
  • 15
    • 0029830432 scopus 로고    scopus 로고
    • Gene transfer to hematopoietic cells
    • Brenner MK. Gene transfer to hematopoietic cells. New Engl J Med 1996; 335: 337-339.
    • (1996) New Engl J Med , vol.335 , pp. 337-339
    • Brenner, M.K.1
  • 17
    • 0026512501 scopus 로고
    • Autologous transplantation of canine long-term marrow culture cells genetically marked by retroviral vectors
    • Carter RF et al. Autologous transplantation of canine long-term marrow culture cells genetically marked by retroviral vectors. Blood 1992; 79: 356-364.
    • (1992) Blood , vol.79 , pp. 356-364
    • Carter, R.F.1
  • 18
    • 0028158266 scopus 로고
    • Gene transfer into hematopoietic stem cells: Long-term maintenance of in vitro activated progenitors without marrow ablation
    • Bienzle D et al. Gene transfer into hematopoietic stem cells: long-term maintenance of in vitro activated progenitors without marrow ablation. Proc Natl Acad Sci USA 1994; 91: 350-354.
    • (1994) Proc Natl Acad Sci USA , vol.91 , pp. 350-354
    • Bienzle, D.1
  • 19
    • 0030952234 scopus 로고    scopus 로고
    • A murine model for human cord blood transplantation: Near-term fetal and neonatal peripheral blood cells can achieve long-term bone marrow engraftment in sublethally irradiated adult recipients
    • Scaradavou A et al. A murine model for human cord blood transplantation: near-term fetal and neonatal peripheral blood cells can achieve long-term bone marrow engraftment in sublethally irradiated adult recipients. Blood 1998; 89: 1089-1099.
    • (1998) Blood , vol.89 , pp. 1089-1099
    • Scaradavou, A.1
  • 20
    • 0030016887 scopus 로고    scopus 로고
    • Arylsulfatase B activities and glycosaminoglycan levels in retrovirally transduced mucopolysaccharidosis VI cells: Prospects for gene therapy
    • Fillat C et al. Arylsulfatase B activities and glycosaminoglycan levels in retrovirally transduced mucopolysaccharidosis VI cells: prospects for gene therapy. J Clin Invest 1996; 98: 497-502.
    • (1996) J Clin Invest , vol.98 , pp. 497-502
    • Fillat, C.1
  • 21
    • 0030791383 scopus 로고    scopus 로고
    • Enzyme replacement therapy in mucopolysaccharidosis VI: Evidence for immune responses and altered efficacy of treatment in animal models
    • Brooks DA et al. Enzyme replacement therapy in mucopolysaccharidosis VI: evidence for immune responses and altered efficacy of treatment in animal models. Biochem Biophys Acta 1997; 1361: 203-216.
    • (1997) Biochem Biophys Acta , vol.1361 , pp. 203-216
    • Brooks, D.A.1
  • 22
    • 0028926009 scopus 로고
    • Myelosuppressive conditioning improves autologous engraftment of genetically marked hematopoietic repopulating cells in dogs
    • Barquinero J et al. Myelosuppressive conditioning improves autologous engraftment of genetically marked hematopoietic repopulating cells in dogs. Blood 1995; 85: 1195-1201.
    • (1995) Blood , vol.85 , pp. 1195-1201
    • Barquinero, J.1
  • 23
    • 0028597587 scopus 로고
    • The use of umbilical cord blood in mismatched related and unrelated hemopoietic stem cell transplantation
    • Kurtzberg J et al. The use of umbilical cord blood in mismatched related and unrelated hemopoietic stem cell transplantation. Blood Cells 1994; 20: 275-283.
    • (1994) Blood Cells , vol.20 , pp. 275-283
    • Kurtzberg, J.1
  • 24
    • 0031984855 scopus 로고    scopus 로고
    • Retroviral gene transfer into cord blood stem/progenitor cells using purified vector stocks
    • Asch J et al. Retroviral gene transfer into cord blood stem/progenitor cells using purified vector stocks. Am J Hematol 1998; 57: 16-23.
    • (1998) Am J Hematol , vol.57 , pp. 16-23
    • Asch, J.1
  • 25
    • 0028864561 scopus 로고
    • Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency
    • Kohn D et al. Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency. Nature Med 1995; 1: 1017-1023.
    • (1995) Nature Med , vol.1 , pp. 1017-1023
    • Kohn, D.1
  • 26
    • 9244243165 scopus 로고    scopus 로고
    • Bone marrow gene transfer in three patients with adenosine deaminase deficiency
    • Hoogerbrugge PM et al. Bone marrow gene transfer in three patients with adenosine deaminase deficiency. Gene Therapy 1996; 3: 179-183.
    • (1996) Gene Therapy , vol.3 , pp. 179-183
    • Hoogerbrugge, P.M.1
  • 27
    • 0016681625 scopus 로고
    • Arylsulfatase B deficiency in Maroteaux-Lamy syndrome: Cellular studies and carrier identification
    • Beratis NG, Turner BM, Weiss R, Hirshhorn K. Arylsulfatase B deficiency in Maroteaux-Lamy syndrome: cellular studies and carrier identification. Pediatr Res 1975; 9: 475-480.
    • (1975) Pediatr Res , vol.9 , pp. 475-480
    • Beratis, N.G.1    Turner, B.M.2    Weiss, R.3    Hirshhorn, K.4
  • 28
    • 0030734644 scopus 로고    scopus 로고
    • Retrovirus-mediated transfer of the human α-iduronidase cDNA into human hematopoietic progenitor cells leads to correction in frans of Hurler fibroblasts
    • Huang MH et al. Retrovirus-mediated transfer of the human α-iduronidase cDNA into human hematopoietic progenitor cells leads to correction in frans of Hurler fibroblasts. Gene Therapy 1997; 4: 1150-1159.
    • (1997) Gene Therapy , vol.4 , pp. 1150-1159
    • Huang, M.H.1


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.