Gene therapy for severe combined immunodeficiency: are we there yet?

被引：144
作者：
Cavazzana-Calvo, Marina [1 ]
Fischer, Alain
机构：
[1] Hop Necker Enfants Malad, AP HP, Dept Biotehrapie, F-75015 Paris, France
[2] Hop Necker Enfants Malad, AP HP, Dept Biotehrapie, F-75015 Paris, France
[3] Univ Paris 05, Paris, France
[4] INSERM U768, Paris, France
来源：
JOURNAL OF CLINICAL INVESTIGATION | 2007年 / 117卷 / 06期
关键词：
D O I：
10.1172/JCI30953
中图分类号：
R-3 [医学研究方法]; R3 [基础医学];
学科分类号：
1001 ;
摘要：
Inherited and acquired diseases of the hematopoietic system can be cured by allogeneic hematopoietic stem cell transplantation. This treatment strategy is highly successful when an HLA-matched sibling donor is available, but if not, few therapeutic options exist. Gene-modified, autologous bone marrow transplantation can circumvent the severe immunological complications that occur when a related HLA-mismatched donor is used and thus represents an attractive alternative. in therapy this review, we summarize the advantages and limitations associated with the use of gene to cure SCID. Insertional mutagenesis and technological improvements aimed at increasing the safety of this strategy are also discussed.
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页码：1456 / 1465
页数：10
相关论文
共 119 条
  • [1] Acland GM, 2001, NAT GENET, V28, P92, DOI 10.1038/88327
  • [2] Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning
    Aiuti, A
    Slavin, S
    Aker, M
    Ficara, F
    Deola, S
    Mortellaro, A
    Morecki, S
    Andolfi, G
    Tabucchi, A
    Carlucci, F
    Marinello, E
    Cattaneo, F
    Vai, S
    Servida, P
    Miniero, R
    Roncarolo, MG
    Bordignon, C
    [J]. SCIENCE, 2002, 296 (5577) : 2410 - 2413
  • [3] Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies:: report of the European experience 1968-99
    Antoine, C
    Müller, S
    Cant, A
    Cavazzana-Calvo, M
    Veys, P
    Vossen, J
    Fasth, A
    Heilmann, C
    Wulffraat, N
    Seger, R
    Blanche, S
    Friedrich, W
    Abinun, M
    Davies, G
    Bredius, R
    Schulz, A
    Landais, P
    Fischer, A
    [J]. LANCET, 2003, 361 (9357) : 553 - 560
  • [4] Role of the nonspecific DNA-binding region and α helices within the core domain of retroviral integrase in selecting target DNA sites for integration
    Appa, RS
    Shin, CG
    Lee, P
    Chow, SA
    [J]. JOURNAL OF BIOLOGICAL CHEMISTRY, 2001, 276 (49) : 45848 - 45855
  • [5] PRIMARY IMMUNODEFICIENCY CAUSED BY MUTATIONS IN THE GENE ENCODING THE CD3-GAMMA SUBUNIT OF THE LYMPHOCYTE-T RECEPTOR
    ARNAIZVILLENA, A
    TIMON, M
    CORELL, A
    PEREZACIEGO, P
    MARTINVILLA, JM
    REGUEIRO, JR
    [J]. NEW ENGLAND JOURNAL OF MEDICINE, 1992, 327 (08) : 529 - 533
  • [6] Cytotoxic immune response blunts long-term transgene expression after efficient retroviral-mediated hepatic gene transfer in rat
    Aubert, D
    Ménoret, S
    Chiari, E
    Pichard, V
    Durand, S
    Tesson, L
    Moullier, P
    Anegon, I
    Ferry, N
    [J]. MOLECULAR THERAPY, 2002, 5 (04) : 388 - 396
  • [7] BACH FH, 1968, LANCET, V2, P1364
  • [8] Gene therapy progress and prospects: the eye
    Bainbridge, J. W. B.
    Tan, M. H.
    Ali, R. R.
    [J]. GENE THERAPY, 2006, 13 (16) : 1191 - 1197
  • [9] A phase I/II clinical trial of β-globin gene therapy for β-thalassemia.
    Bank, A
    Dorazio, R
    Leboulch, P
    [J]. COOLEY'S ANEMIA EIGHTH SYMPOSIUM, 2005, 1054 : 308 - 316
  • [10] Influence of severe combined immunodeficiency phenotype on the outcome of HLA non-identical, T-cell-depleted bone marrow transplantation - A retrospective European survey from the European Group for Bone Marrow Transplantation and the European Society for Immunodeficiency
    Bertrand, Y
    Landais, P
    Friedrich, W
    Gerritsen, B
    Morgan, G
    Fasth, A
    Cavazzana-Calvo, M
    Porta, F
    Cant, A
    Espanol, T
    Müller, S
    Veys, P
    Vossen, J
    Haddad, E
    Fischer, A
    [J]. JOURNAL OF PEDIATRICS, 1999, 134 (06) : 740 - 748
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