Hematopoietic stem cell gene therapy: Progress on the clinical front

Human Gene Therapy

Volumn 25, Issue 3, 2014, Pages 165-170

  Cavazzana, Marina ^a,b  

^a IMAGINE INSTITUTE   (France)

^b ASSISTANCE PUBLIQUE HÔPITAUX DE PARIS   (France)

Author keywords

[No Author keywords available]

Indexed keywords

AUTOTRANSPLANTATION; EVENT FREE SURVIVAL; GENE THERAPY; GENOTOXICITY; HEMATOPOIESIS; HEMATOPOIETIC STEM CELL GENE THERAPY; HEMATOPOIETIC SYSTEM; HUMAN; HUMAN IMMUNODEFICIENCY VIRUS 1 INFECTION; HUMAN IMMUNODEFICIENCY VIRUS INFECTED PATIENT; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; MYELOABLATIVE CONDITIONING; NONHUMAN; OVERALL SURVIVAL; REVIEW; TARGET CELL;

ANIMALS; CELL- AND TISSUE-BASED THERAPY; GENETIC THERAPY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMATOPOIETIC STEM CELLS; HUMANS;

EID: 84896358329     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2014.2504     Document Type: Review

References (38)

1. Aiuti, A., Cattaneo, F., Galimberti, S., et al. (2009). Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N. Engl. J. Med. 360, 447-458.
2. Allers, K., Hutter, G., Hofmann, J., et al. (2011). Evidence for the cure of HIV infection by CCR5Delta32/Delta32 stem cell transplantation. Blood 117, 2791-2799.
3. Auerbach, A.D. (2009). Fanconi anemia and its diagnosis. Mutat. Res. 668, 4-10.
4. Avedillo Diez, I., Zychlinski, D., Coci, E.G., et al. (2011). Development of novel efficient SIN vectors with improved safety features for Wiskott-Aldrich syndrome stem cell based gene therapy. Mol. Pharm. 8, 1525-1537.
5. Awong, G., Herer, E., Surh, C.D., et al. (2009). Characterization in vitro and engraftment potential in vivo of human progenitor T cells generated from hematopoietic stem cells. Blood 114, 972-982.
6. Berger, R., Bernheim, A., Gluckman, E., and Gisselbrecht, C. (1980). In vitro effect of cyclophosphamide metabolites on chromosomes of Fanconi anaemia patients. Br. J. Haematol. 45, 565-568.
7. Biffi, A., Bartolomae, C.C., Cesana, D., et al. (2011). Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not onco-genic selection. Blood 117, 5332-5339.
8. Biffi, A., Montini, E., Lorioli, L., et al. (2013). Lentiviral he-matopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science 341, 1233158.
9. Cartier, N., Hacein-Bey-Abina, S., Bartholomae, C.C., et al. (2009). Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 326, 818-823.
10. Cavazzana-Calvo, M., Payen, E., Negre, O., et al. (2010). Transfusion independence and HMGA2 activation after gene therapy of human beta-thalassaemia. Nature 467, 318-322.
11. Cieri, N., Mastaglio, S., Oliveira, G., et al. (2014). Adoptive immunotherapy with genetically modified lymphocytes in allogeneic stem cell transplantation. Immunol. Rev. 257, 165-180.
12. Czechowicz, A., Kraft, D., Weissman, I.L., and Bhattacharya, D. (2007). Efficient transplantation via antibody-based clearance of hematopoietic stem cell niches. Science 318, 1296-1299.
13. Fernandes, J.F., Rocha, V., Labopin, M., et al. (2012). Transplantation in patients with SCID: mismatched related stem cells or unrelated cord blood? Blood 119, 2949-2955.
14. Fischer, A., Friedrich, W., Fasth, A., et al. (1991). Reduction of graft failure by a monoclonal antibody (anti-LFA-1 CD11a) after HLA nonidentical bone marrow transplantation in children with immunodeficiencies, osteopetrosis, and Fanco-ni's anemia: a European Group for Immunodeficiency/European Group for Bone Marrow Transplantation report. Blood 77, 249-256.
15. Gaspar, H.B., Cooray, S., Gilmour, K.C., et al. (2011). Hema-topoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction. Sci. Transl. Med. 3, 97ra80.
16. Gennery, A.R., Slatter, M.A., Grandin, L., et al. (2010). 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. 126, 602-610.e1-11.
17. Hacein-Bey-Abina, S., Von Kalle, C., Schmidt, M., et al. (2003). LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302, 415-419.
18. Hacein-Bey-Abina, S., Garrigue, A., Wang, G.P., et al. (2008). Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J. Clin. Invest. 118, 3132-3142.
19. Hacein-Bey-Abina, S., Hauer, J., Lim, A., et al. (2010). Efficacy of gene therapy for X-linked severe combined immunodeficiency. N. Engl. J. Med. 363, 355-364.
20. Howe, S.J., Mansour, M.R., Schwarzwaelder, K., et al. (2008). Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. J. Clin. Invest. 118, 3143-3150.
21. Hutter, G., Nowak, D., Mossner, M., et al. (2009). Long-term control of HIV by CCR5 Delta32/Delta32 stem-cell transplantation. N. Engl. J. Med. 360, 692-698.
22. Kustikova, O., Fehse, B., Modlich, U., et al. (2005). Clonal dominance of hematopoietic stem cells triggered by retroviral gene marking. Science 308, 1171-1174.
23. Lagresle-Peyrou, C., Benjelloun, F., Hue, C., et al. (2008). Restoration of human B-cell differentiation into NOD-SCID mice engrafted with gene-corrected CD34+ cells isolated from Artemis or RAG1-deficient patients. Mol. Ther. 16, 396-403.
24. Moiani, A., Miccio, A., Rizzi, E., et al. (2013). Deletion of the LTR enhancer/promoter has no impact on the integration profile of MLV vectors in human hematopoietic progenitors. PLoS One 8, e55721.
25. Montini, E., Cesana, D., Schmidt, M., et al. (2009). The gen-otoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy. J. Clin. Invest. 119, 964-975.
26. Moratto, D., Giliani, S., Bonfim, C., et al. (2011). Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study. Blood 118, 1675-1684.
27. Naldini, L., Blomer, U., Gallay, P., et al. (1996). In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector [see comments]. Science 272, 263-267.
28. Noordzij, J.G., De Bruin-Versteeg, S., Verkaik, N.S., et al. (2002). The immunophenotypic and immunogenotypic B-cell differentiation arrest in bone marrow of RAG-deficient SCID patients corresponds to residual recombination activities of mutated RAG proteins. Blood 100, 2145-2152.
29. Noordzij, J.G., Verkaik, N.S., Van Der Burg, M., et al. (2003). Radiosensitive SCID patients with Artemis gene mutations show a complete B-cell differentiation arrest at the pre-B-cell receptor checkpoint in bone marrow. Blood 101, 1446-1452.
30. Pawliuk, R., Westerman, K.A., Fabry, M.E., et al. (2001). Correction of sickle cell disease in transgenic mouse models by gene therapy. Science 294, 2368-2371.
31. Reimann, C., Six, E., Dal-Cortivo, L., et al. (2012). Human T-lymphoid progenitors generated in a feeder-cell-free Delta-like-4 culture system promote T-cell reconstitution in NOD/SCID/gammac(-/-) mice. Stem Cells 30, 1771-1780.
32. Sauce, D., Larsen, M., Fastenackels, S., et al. (2011). HIV disease progression despite suppression of viral replication is associated with exhaustion of lymphopoiesis. Blood 117, 5142-5151.
33. Schmidt, M., Hoffmann, G., Wissler, M., et al. (2001). Detection and direct genomic sequencing of multiple rare unknown flanking DNA in highly complex samples. Hum. Gene Ther. 12, 743-749.
34. Schroder, A.R., Shinn, P., Chen, H., et al. (2002). HIV-1 integration in the human genome favors active genes and local hotspots. Cell 110, 521-529.
35. Socie, G., Devergie, A., Girinski, T., et al. (1998). Transplantation for Fanconi's anaemia: long-term follow-up of fifty patients transplanted from a sibling donor after low-dose cyclophosphamide and thoraco-abdominal irradiation for conditioning. Br. J. Haematol. 103, 249-255.
36. Stein, S., Ott, M.G., Schultze-Strasser, S., et al. (2010). Geno-mic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease. Nat. Med. 16, 198-204.
37. Wang, G.P., Berry, C.C., Malani, N., et al. (2010). Dynamics of gene-modified progenitor cells analyzed by tracking retroviral integration sites in a human SCID-X1 gene therapy trial. Blood 115, 4356-4366.
38. Yukl, S.A., Boritz, E., Busch, M., et al. (2013). Challenges in detecting HIV persistence during potentially curative interventions: a study of the Berlin patient. PLoS Pathog. 9, e1003347.