Successful Reconstitution of Immunity in ADA-SCID by Stem Cell Gene Therapy Following Cessation of PEG-ADA and Use of Mild Preconditioning

Molecular Therapy

Volumn 14, Issue 4, 2006, Pages 505-513

  Gaspar, H Bobby ^a,b   Bjorkegren, Emma ^a   Parsley, Kate ^a,b   Gilmour, Kimberly C ^a,b   King, Doug ^a   Sinclair, Joanna ^a   Zhang, Fang ^a   Giannakopoulos, Aris ^a   Adams, Stuart ^c   Fairbanks, Lynette D ^d   Gaspar, Jane ^b   Henderson, Lesley ^b   Xu Bayford, Jin Hua ^b   Davies, E Graham ^b   Veys, Paul A ^c   Kinnon, Christine ^a   Thrasher, Adrian J ^a,b  

^a UNIVERSITY COLLEGE LONDON   (United Kingdom)

^b Department of Clinical Immunology ^*  (United Kingdom)

^c Great Ormond Street Hospital for Children NHS Trust   (United Kingdom)

^d GUY'S AND ST THOMAS' NHS FOUNDATION TRUST   (United Kingdom)

Author keywords

adenosine deaminase; gene therapy; retroviral vector; SCID

Indexed keywords

ADENOSINE DEAMINASE; CD34 ANTIGEN; MELPHALAN; RETROVIRUS VECTOR;

ARTICLE; AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION; CASE REPORT; CELL LINEAGE; COMBINED IMMUNODEFICIENCY; CYTOPENIA; ENZYME ACTIVITY; ENZYME DEFICIENCY; ENZYME REPLACEMENT; ERYTHROCYTE; GENETIC TRANSDUCTION; HEMATOPOIESIS; HUMAN; HUMAN CELL; IMMUNITY; LYMPHOCYTE COUNT; NONHUMAN; STEM CELL GENE THERAPY;

ADENOSINE DEAMINASE; CELL LINEAGE; CELLS, CULTURED; CHILD, PRESCHOOL; FOLLOW-UP STUDIES; GENE DOSAGE; GENE THERAPY; GENETIC VECTORS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMATOPOIETIC STEM CELLS; HUMANS; INFANT; MALE; POLYETHYLENE GLYCOLS; SEVERE COMBINED IMMUNODEFICIENCY; T-LYMPHOCYTES; TRANSPLANTATION CONDITIONING;

EID: 33748413936     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ymthe.2006.06.007     Document Type: Article

References (25)

1. Antoine C., et al. Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968-99. Lancet 361 (2003) 553-560
2. Hershfield M.S. PEG-ADA: an alternative to haploidentical bone marrow transplantation and an adjunct to gene therapy for adenosine deaminase deficiency. Hum. Mutat. 5 (1995) 107-112
3. Chan B., et al. Long term efficacy of enzyme replacement therapy for adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID). Clin. Immunol. 117 (2005) 133-143
4. Malacarne F., et al. Reduced thymic output, increased spontaneous apoptosis and oligoclonal B cells in polyethylene glycol-adenosine deaminase-treated patients. Eur.J. Immunol. 35 (2005) 3376-3386
5. Kaufman D.A., Hershfield M.S., Bocchini J.A., Moissidis I.J., Jeroudi M., and Bahna S.L. Cerebral lymphoma in an adenosine deaminase-deficient patient with severe combined immunodeficiency receiving polyethylene glycol-conjugated adenosine deaminase. Pediatrics 116 (2005) e876-e879
6. Bordignon C., et al. Gene therapy in peripheral blood lymphocytes and bone marrow for ADA-immunodeficient patients. Science 270 (1995) 470-475
7. Kohn D.B., et al. Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency. Nat. Med. 1 (1995) 1017-1023
8. Muul L.M. Persistence and expression of the adenosine deaminase gene for 12 years and immune reaction to gene transfer components: long-term results of the first clinical gene therapy trial. Blood 101 (2003) 2563-2569
9. + cells from the cord blood of ADA-deficient SCID neonates. Nat. Med. 9 (2003) 463-468
10. Aiuti A., et al. Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement. Nat. Med. 8 (2002) 423-425
11. Aiuti A., et al. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 296 (2002) 2410-2413
12. Hacein-Bey-Abina S., et al. Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N. Engl. J. Med. 346 (2002) 1185-1193
13. Gaspar H.B., et al. Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet 364 (2004) 2181-2187
14. Otsu M., et al. Generalised detoxification associated with engraftment of gene-corrected repopulating cells achieved in ADA-SCID patients by stem cell gene therapy without myelopreparative pre-conditioning. Mol. Ther. 11 (2005) S14-S15
15. Rao K., et al. Improved survival after unrelated donor bone marrow transplantation in children with primary immunodeficiency using a reduced-intensity conditioning regimen. Blood 105 (2005) 879-885
16. Demaison C., et al. High-level transduction and gene expression in hematopoietic repopulating cells using a human immunodeficiency virus type 1-based lentiviral vector containing an internal spleen focus forming virus promoter. Hum. Gene Ther. 13 (2002) 803-813
17. Thrasher A.J., et al. Failure of SCID-X1 gene therapy in older patients. Blood 105 (2005) 4255-4257
18. Li Z., et al. Murine leukemia induced by retroviral gene marking. Science 296 (2002) 497
19. Baum C., et al. Chance or necessity? Insertional mutagenesis in gene therapy and its consequences. Mol. Ther. 9 (2004) 5-13
20. Hacein-Bey-Abina S., et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302 (2003) 415-419
21. Themis M., et al. Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal mice. Mol. Ther. 12 (2005) 763-771
22. Kingsman S.M., Mitrophanous K., and Olsen J.C. Potential oncogene activity of the woodchuck hepatitis post-transcriptional regulatory element (WPRE). Gene Ther. 12 (2005) 3-4
23. Zufferey R., Donello J.E., Trono D., and Hope T.J. Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors. J. Virol. 73 (1999) 2886-2892
24. Otsu M., et al. Flow cytometry analysis of adenosine deaminase (ADA) expression: a simple and reliable tool for the assessment of ADA-deficient patients before and after gene therapy. Hum. Gene Ther. 13 (2002) 425-432
25. King D.J., Gotch F.M., and Larsson-Sciard E.L. T-cell re-population in HIV-infected children on highly active anti-retroviral therapy (HAART). Clin. Exp. Immunol. 125 (2001) 447-454