Gene therapy for primary adaptive immune deficiencies

Journal of Allergy and Clinical Immunology

Volumn 127, Issue 6, 2011, Pages 1356-1359

  Fischer, Alain ^a,b,c   Hacein Bey Abina, Salima ^a,b,c,d   Cavazzana Calvo, Marina ^a,b,c,d  

^a INSERM   (France)

^b UNIVERSITÉ PARIS DESCARTES   (France)

^c HÔPITAL NECKER ENFANTS MALADES   (France)

^d ASSISTANCE PUBLIQUE HÔPITAUX DE PARIS   (France)

Author keywords

Gene therapy; lentivirus; retrovirus; severe combined immune deficiency; Wiskott Aldrich syndrome

Indexed keywords

ADENOSINE DEAMINASE; BUSULFAN; CD34 ANTIGEN; LENTIVIRUS VECTOR; LIM DOMAIN ONLY 2; LIM PROTEIN; RETROVIRUS VECTOR; T LYMPHOCYTE RECEPTOR; UNCLASSIFIED DRUG; WISKOTT ALDRICH SYNDROME PROTEIN;

ADENOSINE DEAMINASE DEFICIENCY; ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; B LYMPHOCYTE; CANCER CHEMOTHERAPY; CELL FUNCTION; CELLULAR IMMUNITY; DRUG EFFICACY; ENGRAFTMENT; ENZYME REPLACEMENT; GENE THERAPY; GENE TRANSFER; GENOTOXICITY; HUMAN; LENTIVIRINAE; LEUKEMIA; LONG TERMINAL REPEAT; NATURAL KILLER CELL; PRE T LYMPHOCYTE; PRIORITY JOURNAL; PROTO ONCOGENE; RETROVIRUS; REVIEW; SPUMA VIRUS; STEM CELL; T CELL LEUKEMIA; T LYMPHOCYTE; THROMBOCYTOPENIA; TRANSGENE; TRANSPOSON; VIRAL GENE DELIVERY SYSTEM; WISKOTT ALDRICH SYNDROME; X LINKED SEVERE COMBINED IMMUNODEFICIENCY;

ADAPTIVE IMMUNITY; ADENOSINE DEAMINASE; AGAMMAGLOBULINEMIA; CLINICAL TRIALS AS TOPIC; GENE THERAPY; GENETIC VECTORS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; IMMUNOLOGIC DEFICIENCY SYNDROMES; SEVERE COMBINED IMMUNODEFICIENCY; TREATMENT OUTCOME; WISKOTT-ALDRICH SYNDROME; X-LINKED COMBINED IMMUNODEFICIENCY DISEASES;

EID: 79957809524     PISSN: 00916749     EISSN: 10976825     Source Type: Journal    
DOI: 10.1016/j.jaci.2011.04.030     Document Type: Review

References (33)

1. Gennery AR, Slatter MA, Grandin L, Taupin P, Cant AJ, Veys P, et al. Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: entering a new century, do we do better? J Allergy Clin Immunol 2010;126:602-10, e1-11.
2. Notarangelo LD, Fischer A, Geha RS, Casanova JL, Chapel H, Conley ME, et al. Primary immunodeficiencies: 2009 update. J Allergy Clin Immunol 2009;124:1161-78.
3. Verma IM, Weitzman MD. Gene therapy: twenty-first century medicine. Annu Rev Biochem 2005;74:711-38.
4. Yu SF, von Ruden T, Kantoff PW, Garber C, Seiberg M, Ruther U, et al. Self-inactivating retroviral vectors designed for transfer of whole genes into mammalian cells. Proc Natl Acad Sci U S A 1986;83:3194-8. (Pubitemid 16043003)
5. Cavazzana-Calvo M, Hacein-Bey S, De Saint Basile G, Gross F, Yvon E, Nusbaum P, et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 2000;288:669-72. (Pubitemid 30241569)
6. Hacein-Bey-Abina S, Hauer J, Lim A, Picard C, Wang GP, Berry CC, et al. Efficacy of gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 2010;363:355-64.
7. Gaspar HB, Parsley KL, Howe S, King D, Gilmour KC, Sinclair J, et al. Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet 2004;364:2181-7. (Pubitemid 40018447)
8. Aiuti A, Slavin S, Aker M, Ficara F, Deola S, Mortellaro A, et al. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 2002;296:2410-3. (Pubitemid 34734219)
9. Aiuti A, Cattaneo F, Galimberti S, Benninghoff U, Cassani B, Callegaro L, et al. Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N Engl J Med 2009;360:447-58.
10. Hacein-Bey-Abina S, Garrigue A, Wang GP, Soulier J, Lim A, Morillon E, et al. Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J Clin Invest 2008;118:3132-42.
11. Howe SJ, Mansour MR, Schwarzwaelder K, Bartholomae C, Hubank M, Kempski H, et al. Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. J Clin Invest 2008; 118:3143-50.
12. Dave UP, Akagi K, Tripathi R, Cleveland SM, Thompson MA, Yi M, et al. Murine leukemias with retroviral insertions at Lmo2 are predictive of the leukemias induced in SCID-X1 patients following retroviral gene therapy. PLoS Genet 2009;5: e1000491.
13. Santoni FA, Hartley O, Luban J. Deciphering the code for retroviral integration target site selection. PLoS Comput Biol 2010;6:e1001008.
14. Ferrua F, Brigida I, Aiuti A. Update on gene therapy for adenosine deaminase-deficient severe combined immunodeficiency. Curr Opin Allergy Clin Immunol 2010;10:551-6.
15. Cattoglio C, Pellin D, Rizzi E, Maruggi G, Corti G, Miselli F, et al. High-definition mapping of retroviral integration sites identifies active regulatory elements in human multipotent hematopoietic progenitors. Blood 2010;116:5507-17.
16. Kustikova O, Brugman M, Baum C. The genomic risk of somatic gene therapy. Semin Cancer Biol 2010;20:269-78.
17. Papapetrou EP, Lee G, Malani N, Setty M, Riviere I, Tirunagari LM, et al. Genomic safe harbors permit high beta-globin transgene expression in thalassemia induced pluripotent stem cells. Nat Biotechnol 2011;29:73-8.
18. Lombardo A, Genovese P, Beausejour CM, Colleoni S, Lee YL, Kim KA, et al. Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery. Nat Biotechnol 2007;25:1298-306. (Pubitemid 350076515)
19. Thrasher AJ, Hacein-Bey-Abina S, Gaspar HB, Blanche S, Davies EG, Parsley K, et al. Failure of SCID-X1 gene therapy in older patients. Blood 2005;105:4255-7. (Pubitemid 40720770)
20. Gaspar HB, Aiuti A, Porta F, Candotti F, Hershfield MS, Notarangelo LD. How I treat ADA deficiency. Blood 2009;114:3524-32.
21. Cartier N, Hacein-Bey-Abina S, Bartholomae CC, Veres G, Schmidt M, Kutschera I, et al. Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 2009;326:818-23.
22. Boztug K, Schmidt M,Schwarzer A, Banerjee PP, Diez IA,Dewey RA, et al. Stem-cell gene therapy for theWiskott-Aldrich syndrome. N Engl J Med 2010;363:1918-27.
23. Benjelloun F, Garrigue A, Demerens-de Chappedelaine C, Soulas-Sprauel P, Malassis-Seris M, Stockholm D, et al. Stable and functional lymphoid reconstitution in Artemis-deficient mice following lentiviral Artemis gene transfer into hematopoietic stem cells. Mol Ther 2008;16:1490-9.
24. Mostoslavsky G, Fabian AJ, Rooney S, Alt FW, Mulligan RC. Complete correction of murine Artemis immunodeficiency by lentiviral vector-mediated gene transfer. Proc Natl Acad Sci U S A 2006;103:16406-11. (Pubitemid 44715209)
25. Lagresle-Peyrou C, Yates F, Malassis-Seris M, Hue C, Morillon E, Garrigue A, et al. Long-term immune reconstitution inRAG-1-deficient mice treated by retroviral gene therapy: a balance between efficiency and toxicity. Blood 2006;107:63-72. (Pubitemid 43053525)
26. Fischer A, Hacein-Bey-Abina S, Cavazanna-Calvo M. Gene therapy for primary immunodeficiencies. Immunol Allergy Clin North Am 2010;30:237-48.
27. Grez M, Reichenbach J, Schwable J, Seger R, Dinauer MC, Thrasher AJ. Gene therapy of chronic granulomatous disease: the engraftment dilemma. Mol Ther 2011;19:28-35.
28. Hunter MJ, Tuschong LM, Fowler CJ, Bauer TR Jr, Burkholder TH, Hickstein DD. Gene therapy of canine leukocyte adhesion deficiency using lentiviral vectors with human CD11b and CD18 promoters driving canine CD18 expression. Mol Ther 2011;19:113-21.
29. Brown MP, Topham DJ, Sangster MY, Zhao J, Flynn KJ, Surman SL, et al. Thymic lymphoproliferative disease after successful correction of CD40 ligand deficiency by gene transfer in mice. Nat Med 1998;4:1253-60. (Pubitemid 28512105)
30. Zhang F, Cong L, Lodato S, Kosuri S, Church GM, Arlotta P. Efficient construction of sequence-specific TAL effectors for modulating mammalian transcription. Nat Biotechnol 2011;29:149-53.
31. Urnov FD, Rebar EJ, Holmes MC, Zhang HS, Gregory PD. Genome editing with engineered zinc finger nucleases. Nat Rev Genet 2010;11:636-46.
32. Boitano AE, Wang J, Romeo R, Bouchez LC, Parker AE, Sutton SE, et al. Aryl hydrocarbon receptor antagonists promote the expansion of human hematopoietic stem cells. Science 2010;329:1345-8.
33. Szabo E, Rampalli S, Risueno RM, Schnerch A, Mitchell R, Fiebig-Comyn A, et al. Direct conversion of human fibroblasts to multilineage blood progenitors. Nature 2010;468:521-6.