Critical Variables affecting clinical-grade production of the self-inactivating gamma-retroviral vector for the treatment of X-linked severe combined immunodeficiency

Gene Therapy

Volumn 19, Issue 8, 2012, Pages 872-876

  Van Der Loo, J C M ^a   Swaney, W P ^b   Grassman, E ^a   Terwilliger, A ^a   Higashimoto, T ^a   Schambach, A ^c   Hacein Bey Abina, S ^d,e,f   Nordling, D L ^a   Cavazzana Calvo, M ^d,e,f   Thrasher, A J ^g,h   Williams, D A ⁱ   Reeves, L ^j   Malik, P ^a  

^a CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER   (United States)

^b USA   (United States)

^c HANNOVER MEDICAL SCHOOL   (Germany)

^d HÔPITAL NECKER ENFANTS MALADES   (France)

^e Clinical Investigation Center in Biotherapy   (France)

^f UNIVERSITÉ PARIS DESCARTES   (France)

^g UNIVERSITY COLLEGE LONDON   (United Kingdom)

^h Great Ormond Street Hospital NHS Trust ^*  (United Kingdom)

ⁱ BOSTON CHILDREN S HOSPITAL   (United States)

^j INDIANA UNIVERSITY SCHOOL OF MEDICINE   (United States)

more..

Author keywords

clinical grade; gamma retrovirus; GMP; process development; SCID X1; self inactivating

Indexed keywords

CD34 ANTIGEN; GLUTAMINE; INTERLEUKIN 2 RECEPTOR GAMMA; RETROVIRUS VECTOR;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; COLONY FORMING UNIT; CONTROLLED STUDY; CULTURE MEDIUM; DISSOCIATION; GENE THERAPY; GENE TRANSFER; GENETIC TRANSFECTION; HUMAN; HUMAN CELL; MOUSE; NONHUMAN; PRIORITY JOURNAL; T LYMPHOCYTE; X LINKED SEVERE COMBINED IMMUNODEFICIENCY; XENOTRANSPLANTATION;

ANIMALS; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS; INTERLEUKIN RECEPTOR COMMON GAMMA SUBUNIT; LEUKEMIA VIRUS, GIBBON APE; MICE; MICE, INBRED NOD; MICE, SCID; RETROVIRIDAE; TERMINAL REPEAT SEQUENCES; TRANSDUCTION, GENETIC; X-LINKED COMBINED IMMUNODEFICIENCY DISEASES;

GIBBON APE LEUKEMIA VIRUS; MUS;

EID: 84864875026     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2012.37     Document Type: Article

References (25)

1. Noguchi M, Yi H, Rosenblatt HM, Filipovich AH, Adelstein S, Modi WS et al. Interleukin-2 receptor gamma chain mutation results in X-linked severe combined immunodeficiency in humans. Cell 1993; 73: 147-157.
2. 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.
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. Williams DA, Baum C. Medicine. Gene therapy-new challenges ahead. Science 2003; 302: 400-401.
7. 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.
8. 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: 3143-3150.
9. Gaspar HB, Cooray S, Gilmour KC, Parsley KL, Adams S, Howe SJ et al. Long-term persistence of a polyclonal T cell repertoire after gene therapy for x-linked severe combined immunodeficiency. Sci Transl Med 2011; 3: 97ra79.
10. McCormack MP, Rabbitts TH. Activation of the T-cell oncogene LMO2 after gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 2004; 350: 913-922.
11. 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: 3132-3142.
12. Baum C, Dullmann J, Li Z, Fehse B, Meyer J, Williams DA et al. Side effects of retroviral gene transfer into hematopoietic stem cells. Blood 2003; 101: 2099-2114.
13. Thornhill SI, Schambach A, Howe SJ, Ulaganathan M, Grassman E, Williams D et al. Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency. Mol Ther 2008; 16: 590-598.
14. Kraunus J, Schaumann DH, Meyer J, Modlich U, Fehse B, Brandenburg G et al. Selfinactivating retroviral vectors with improved RNA processing. Gene Therapy 2004; 11: 1568-1578.
15. Zychlinski D, Schambach A, Modlich U, Maetzig T, Meyer J, Grassman E et al. Physiological Promoters Reduce the Genotoxic Risk of Integrating Gene Vectors. Mol Ther 2008; 16: 718-725.
16. Schambach A, Swaney WS, van der Loo JCM. Design and production of retro-and lentiviral vectors for gene expression in hematopoietic cells. In: Baum C (ed). Method in Mol Biol 506. Humana Press: Totowa, NJ, 2009, pp 191-205.
17. van der Loo JC, Swaney WP, Grassman E, Terwilliger A, Higashimoto T, Schambach A et al. Scale-up and manufacturing of clinical-grade self-inactivating gammaretroviral vectors by transient transfection. Gene Therapy 2012; 19: 246-254.
18. Tritsch GL, Moore GE. Spontaneous decomposition of glutamine in cell culture media. Exp Cell Res 1962; 28: 360-364.
19. Lin A, Agrawal P. Glutamine decomposition in DMEM: effect of pH and serum concentration. Biotechnol Lett 1988; 10: 695-698.
20. Heeneman S, Deutz NE, Buurman WA. The concentrations of glutamine and ammonia in commercially available cell culture media. J Immunol Methods 1993; 166: 85-91.
21. Nordling D, Kaiser A, Reeves L. Release testing of retroviral vectors and gene-modified cells. Methods Mol Biol 2009; 506: 265-279.
22. Schleef M, Schmidt T. Annimal-free production of ccc-supercoiled plasmids for research and clinical application. J Gene Med 2004; 6: S45-S53.
23. Villella AD, Yao J, Getty RR, Juliar BE, Yiannoutsos C, Hartwell JR et al. Real-time PCR: an effective tool for measuring transduction efficiency in human hematopoietic progenitor cells. Mol Ther 2005; 11: 483-491.
24. Sastry L, Xu Y, Cooper R, Pollok K, Cornetta K. Evaluation of plasmid DNA removal from lentiviral vectors by benzonase treatment. Hum Gene Ther 2004; 15: 221-226.
25. De Smedt M, Hoebeke I, Plum J. Human bone marrow CD34 + progenitor cells mature to T cells on OP9-DL1 stromal cell line without thymus microenvironment. Blood Cells Mol Dis 2004; 33: 227-232.