Immunodeficiencies: Injecting some safety into SCID gene therapy?

Gene Therapy

Volumn 13, Issue 9, 2006, Pages 741-743

  Candotti, F ^a   Roifman, C ^b   Puck, J M ^a  

^a NATIONAL HUMAN GENOME RESEARCH INSTITUTE   (United States)

^b HOSPITAL FOR SICK CHILDREN   (Canada)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOSINE DEAMINASE; JANUS KINASE 3; LENTIVIRUS VECTOR; PROTEIN KINASE ZAP 70; RAG1 PROTEIN; RAG2 PROTEIN; RETROVIRUS VECTOR;

B LYMPHOCYTE; CELL BASED GENE THERAPY; CELL DIFFERENTIATION; CELL FUNCTION; CELL LINEAGE; CLINICAL TRIAL; COMBINED IMMUNODEFICIENCY; EX VIVO STUDY; GENE TARGETING; GENE TRANSFER; HUMAN; NATURAL KILLER CELL; NONHUMAN; NOTE; PRIORITY JOURNAL; PROTEIN DEFICIENCY; SCID MOUSE; STEM CELL TRANSPLANTATION; STROMA CELL; T LYMPHOCYTE ACTIVATION; THYMOCYTE; VIRAL GENE DELIVERY SYSTEM;

ANIMALS; GENE THERAPY; GENETIC VECTORS; HUMANS; LENTIVIRUS; MICE; SEVERE COMBINED IMMUNODEFICIENCY; THYMUS GLAND; ZAP-70 PROTEIN-TYROSINE KINASE;

LENTIVIRUS;

EID: 33645995450     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/sj.gt.3302663     Document Type: Note

References (19)

1. 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: 669-672.
2. 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: 2410-2413.
3. Hacein-Bey-Abina S, Le Deist F, Carlier F, Bouneaud C, Hue C, De Villartay JP et al. Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N Engl J Med 2002; 346: 1185-1193.
4. 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: 2181-2187.
5. Hacein-Bey-Abina S, von Kalle C, Schmidt M, Le Deist F, Wulffraat N, McIntyre E et al. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 2003; 348: 255-256.
6. Hacein-Bey-Abina S, Von Kalle C, Schmidt M, McCormack MP, Wulffraat N, Leboulch P et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 2003; 302: 415-419.
7. Wu X, Li Y, Crise B, Burgess SM. Transcription start regions in the human genome are favored targets for MLV integration. Science 2003; 300: 1749-1751.
8. Mitchell RS, Beitzel BF, Schroder AR, Shinn P, Chen H, Berry CC et al. Retroviral DNA integration: ASLV, HIV, and MLV show distinct target site preferences. PLoS Biol 2004; 2: E234.
9. Dull T, Zufferey R, Kelly M, Mandel RJ, Nguyen M, Trono D et al. A third-generation lentivirus vector with a conditional packaging system. J Virol 1998; 72: 8463-8471.
10. Miyoshi H, Blomer U, Takahashi M, Gage FH, Verma IM. Development of a self-inactivating lentivirus vector. J Virol 1998; 72: 8150-8157.
11. Rebel VI, Tanaka M, Lee JS, Hartnett S, Pulsipher M, Nathan DG et al. One-day ex vivo culture allows effective gene transfer into human nonobese diabetic/severe combined immune-deficient repopulating cells using high-titer vesicular stomatitis virus G protein pseudotyped retrovirus. Blood 1999; 93: 2217-2224.
12. Dorrell C, Gan OI, Pereira DS, Hawley RG, Dick JE. Expansion of human cord blood CD34(+)CD38(-) cells in ex vivo culture during retroviral transduction without a corresponding increase in SCID repopulating cell (SRC) frequency: Dissociation of SRC phenotype and function. Blood 2000; 95: 102-110.
13. Adjali O, Vicente RR, Ferrand C, Jacquet C, Mongellaz C, Tiberghien P et al. In vivo correction of ZAP-70 immunodeficiency by intrathymic gene transfer. J Clin Invest 2005; 115: 2287-2295.
14. Roifman CM. Studies of patients' thymi aid in the discovery and characterization of immunodeficiency in humans. Immunol Rev 2005; 203: 143-155.
15. Stephan V, Wahn V, Le Deist F, Dirksen U, Broker B, Muller-Fleckenstein I et al. Atypical X-linked severe combined immunodeficiency due to possible spontaneous reversion of the genetic defect in T cells. N Engl J Med 1996; 335: 1563-1567.
16. Nelson DM, Metzger ME, Donahue RE, Morgan RA. In vivo retrovirus-mediated gene transfer into multiple hematopoietic lineages in rabbits without preconditioning. Hum Gene Ther 1997; 8: 747-754.
17. Newbound GC, Cooper JR, O'Rourke JP, Baskin CR, Bunnell BA. Analysis of gene transfer efficiency of retrovirus producer cell transplantation for in situ gene transfer to hematopoietic cells. Exp Hematol 2001; 29: 163-173.
18. McCauslin CS, Wine J, Cheng L, Klarmann KD, Candotti F, Clausen PA et al. In vivo retroviral gene transfer by direct intrafemoral injection results in correction of the SCID phenotype in JaK3 knock-out animals. Blood 2003; 102: 843-848.
19. Ueda K, Hanazono Y, Shibata H, Ageyama N, Ueda Y, Ogata S et al. High-level in vivo gene marking after gene-modified autologous hematopoietic stem cell transplantation without marrow conditioning in nonhuman primates. Mol Ther 2004; 10: 469-477.