Failure of SCID-X1 gene therapy in older patients-Reference-Cited by-同舟云学术

Failure of SCID-X1 gene therapy in older patients

Published:2005-06-01 Issue:11 Volume:105 Page:4255-4257
ISSN:0006-4971
Container-title:Blood
language:en
Short-container-title:

Author:

Thrasher Adrian J.¹,Hacein-Bey-Abina Salima¹,Gaspar H. Bobby¹,Blanche Stephane¹,Davies E. Graham¹,Parsley Kathryn¹,Gilmour Kimberly¹,King Douglas¹,Howe Steven¹,Sinclair Joanna¹,Hue Christophe¹,Carlier Frédérique¹,von Kalle Christof¹,de Saint Basile Geneviève¹,le Deist Françoise¹,Fischer Alain¹,Cavazzana-Calvo Marina¹

Affiliation:

1. From the Molecular Immunology Unit, Institute of Child Health, London, United Kingdom; Department of Biotherapy, Unité d'Immunologie et d'Hématologie Pédiatriques, Institut National de la Santé et de la Recherche Médicale (INSERM) Unit 429, and Laboratoire d'Immunologie Pédiatrique, Hôpital Necker, Paris, France; Immunology Unit, Great Ormond Street Hospital for Children, London, United Kingdom; and Cincinnati Children's Research Foundation, Cincinnati, OH.

Abstract

AbstractGene therapy has been shown to be a highly effective treatment for infants with typical X-linked severe combined immunodeficiency (SCID-X1, γc-deficiency). For patients in whom previous allogeneic transplantation has failed, and others with attenuated disease who may present later in life, the optimal treatment strategy in the absence of human leukocyte antigen (HLA)–matched donors is unclear. Here we report the failure of gene therapy in 2 such patients, despite effective gene transfer to bone marrow CD34+ cells, suggesting that there are intrinsic host-dependent restrictions to efficacy. In particular, there is likely to be a limitation to initiation of normal thymopoiesis, and we therefore suggest that intervention for these patients should be considered as early as possible.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

Link

http://ashpublications.org/blood/article-pdf/105/11/4255/1708943/zh801105004255.pdf

Reference19 articles.

1. Sugamura K, Asao H, Kondo M, et al. The interleukin-2 receptor gamma chain: its role in the multiple cytokine receptor complexes and T cell development in XSCID. Annu Rev Immunol. 1996;14: 179-205.

2. DiSanto JP, Rieux-Laucat F, Dautry-Varsat A, Fischer A, de Saint Basile G. Defective human interleukin 2 receptor gamma chain in an atypical X chromosome-linked severe combined immunodeficiency with peripheral T cells. Proc Natl Acad Sci U S A. 1994;91: 9466-9470.

3. Buckley RH, Schiff SE, Schiff RI, et al. Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency. N Engl J Med. 1999;340: 508-516.

4. Antoine C, Muller S, Cant A, et al. Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968-99. Lancet. 2003;361: 553-560.

5. Buckley RH. Molecular defects in human severe combined immunodeficiency and approaches to immune reconstitution. Annu Rev Immunol. 2004;22: 625-655.

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