The Role of Conditioning in Hematopoietic Stem-Cell Gene Therapy

Human Gene Therapy

Volumn 27, Issue 10, 2016, Pages 741-748

  Bernardo, Maria Ester ^a   Aiuti, Alessandro ^a,b  

^a SAN RAFFAELE SCIENTIFIC INSTITUTE   (Italy)

^b VITA SALUTE SAN RAFFAELE UNIVERSITY   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

ADRENOLEUKODYSTROPHY; ARTICLE; CHEMOTHERAPY; GENE EXPRESSION; GENE THERAPY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMOGLOBINOPATHY; HUMAN; METACHROMATIC LEUKODYSTROPHY; TRANSGENE; X LINKED SEVERE COMBINED IMMUNODEFICIENCY; ALLOTRANSPLANTATION; CYTOLOGY; HEMATOPOIETIC STEM CELL; PROCEDURES; TRANSPLANTATION CONDITIONING;

GENETIC THERAPY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMATOPOIETIC STEM CELLS; HUMANS; TRANSPLANTATION CONDITIONING; TRANSPLANTATION, HOMOLOGOUS;

EID: 84989246267     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2016.103     Document Type: Article

References (48)

1. Cicalese MP, Aiuti A. Clinical applications of gene therapy for primary immunodeficiencies. Hum Gene Ther 2015;26:210-219.
2. Aiuti A, Biasco L, Scaramuzza S, et al. Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science 2013; 341:1233151.
3. Cartier N, Hacein-Bey-Abina S, Bartholomae CC, et al. Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 2009;326:818-823.
4. Cavazzana-Calvo M, Payen E, Negre O, et al. Transfusion independence and HMGA2 activation after gene therapy of human b-thalassaemia. Nature 2010;467:318-322.
5. Naldini L. Gene therapy returns to centre stage. Nature 2015;526:351-360.
6. Aiuti A, Slavin S, Aker M, et al. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 2002;296:2410-2413.
7. Aiuti A, Cattaneo F, Galimberti S, et al. Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N Engl J Med 2009;360: 447-458.
8. Candotti F, Shaw KL, Muul L, et al. Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans. Blood 2012;120: 3635-3646.
9. Gaspar HB, Cooray S, Gilmour KC, et al. Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction. Sci Transl Med 2011;3:97ra80.
10. Biffi A, Montini E, Lorioli L, et al. Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science 2013;341: 1233158.
11. Hacein-Bey Abina S, Gaspar HB, Blondeau J, et al. Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome. JAMA 2015; 313:1550-1563.
12. Carbonaro DA, Zhang L, Jin X, et al. Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency. Mol Ther 2014;22:607-622.
13. Copelan EA. Hematopoietic stem-cell transplantation. N Engl J Med 2006;354:1813-1826.
14. Bacigalupo A, Ballen K, Rizzo D, et al. Defining the intensity of conditioning regimens: working definitions. Biol Blood Marrow Transplant 2009; 15:1628-1633.
15. Bordignon C, Notarangelo LD, Nobili N, et al. Gene therapy in peripheral blood lymphocytes and bone marrow for ADA-immunodeficient patients. Science 1995;270:470-475.
16. Kohn DB, Weinberg KI, Nolta JA, et al. Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency. Nat Med 1995;1:1017-1023.
17. Biasco L, Ambrosi A, Pellin D, et al. Integration profile of retroviral vector in gene therapy treated patients is cell-specific according to gene expression and chromatin conformation of target cell. EMBO Mol Med 2011;3:89-101.
18. Cicalese MP, Ferrua F, Castagnaro L, et al. Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency. Blood 2016;128:45-54.
19. Biasco L, Scala S, Basso Ricci L, et al. In vivo tracking of T cells in humans unveils decade-long survival and activity of genetically modified T memory stem cells. Sci Transl Med 2015;7: 273ra13.
20. Sauer AV, Mrak E, Hernandez RJ, et al. ADAdeficient SCID is associated with a specific microenvironment and bone phenotype characterized by RANKL/OPG imbalance and osteoblast insufficiency. Blood 2009;114:3216-3226.
21. Sokolic R, Maric I, Kesserwan C, et al. Myeloid dysplasia and bone marrow hypocellularity in adenosine deaminase-deficient severe combined immune deficiency. Blood 2011;118:2688-2694.
22. Hacein-Bey-Abina S, Hauer J, Lim A, et al. Efficacy of gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 2010; 363:355-364.
23. Gaspar HB, Cooray S, Gilmour KC, 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.
24. Cavazzana-Calvo M, Fischer A, Hacein-Bey-Abina S, Aiuti A. Gene therapy for primary immunodeficiencies: Part 1. Curr Opin Immunol 2012;24: 580-584.
25. Hacein-Bey-Abina S, Pai SY, Gaspar HB, et al. A modified c-retrovirus vector for X-linked severe combined immunodeficiency. N Engl J Med 2014;371:1407-1417.
26. Cavazzana M, Six E, Lagresle-Peyrou C, et al. Gene therapy for X-linked severe combined immunodeficiency: where do we stand? Hum Gene Ther 2016;27:108-116.
27. De Ravin SS, Malech HL. Partially corrected Xlinked severe combined immunodeficiency: long-term problems and treatment options. Immunol Res 2009;43:223-242.
28. De Ravin SS, Wu X, Moir S, et al. Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency. Sci Transl Med 2016;8:335ra57.
29. Boztug K, Schmidt M, Schwarzer A, et al. Stemcell gene therapy for the Wiskott-Aldrich syndrome. N Engl J Med 2010;363:1918-1927.
30. Braun CJ, Boztug K, Paruzynski A, et al. Gene therapy for Wiskott-Aldrich syndrome: long-term efficacy and genotoxicity. Sci Transl Med 2014; 6:227ra33.
31. Biasco L, Pellin D, Scala S, et al. In vivo tracking of human hematopoiesis reveals patterns of clonal dynamics during early and steady-state reconstitution phases. Cell Stem Cell 2016; 19:107-119.
32. van Rappard DF, Boelens JJ, van Egmond ME, et al. Efficacy of hematopoietic cell transplantation in metachromatic leukodystrophy: The Dutch experience. Blood 2016;127:3098-3101.
33. Krägeloh-Mann I, Groeschel S, Kehrer C, et al. Juvenile metachromatic leukodystrophy 10 years post transplant compared with a non-transplanted cohort. Bone Marrow Transplant 2013;48:369-375.
34. Capotondo A, Milazzo R, Politi LS, et al. Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantation. Proc Natl Acad Sci U S A 2012;109:15018-15023.
35. Sessa M, Lorioli L, Fumagalli F, et al. Lentiviral haemopoietic stem-cell gene therapy in earlyonset metachromatic leukodystrophy: An ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial. Lancet 2016;388:476-487.
36. Cartier N, Hacein-Bey-Abina S, Bartholomae CC, et al. Lentiviral hematopoietic cell gene therapy for X-linked adrenoleukodystrophy. Methods Enzymol 2012;507:187-198.
37. Cavazzana M, Ribeil JA, Payen E, et al. Study Hgb-205: outcomes of gene therapy for hemoglobinopathies via transplantation of autologous hematopoietic stem cells transduced ex vivo with a lentiviral bA-T87Q-globin vector (LentiGlobin BB305 drug product) [abstract]. Blood 2014;124:4797.
38. Cavazzana M, Ribeil JA, Payen E, et al. Outcomes of gene therapy for severe sickle disease and beta-thalassemia major via transplantation of autologous hematopoietic stem cells transduced ex vivo with a lentiviral beta AT87Q-globin vector. Blood 2015;126: Abstract 202.
39. Bernardo ME, Piras E, Vacca A, et al. Allogeneic hematopoietic stem cell transplantation in thalassemia major: results of a reduced-toxicity conditioning regimen based on the use of treosulfan. Blood 2012;120:473-476.
40. Czechowicz A, Kraft D, Weissman IL, Bhattacharya D. Efficient transplantation via antibodybased clearance of hematopoietic stem cell niches. Science 2007;318:1296-1299.
41. Xue X, Pech NK, Shelley WC, et al. Antibody targeting KIT as pretransplantation conditioning in immunocompetent mice. Blood 2010;116:5419-5422.
42. Straathof K, Rao K, Eyrich M, et al. Haemopoietic stem-cell transplantation with antibody-based minimal-intensity conditioning: A Phase 1/2 study. Lancet 2009;374:912-920.
43. Palchaudhuri R, Saez B, Hoggatt J, et al. Nongenotoxic conditioning for hematopoietic stem cell transplantation using a hematopoietic-cellspecific internalizing immunotoxin. Nat Biotechnol 2016;34:738-745.
44. Hopkins PV, Campbell C, Klug T, et al. Lysosomal storage disorder screening implementation: findings from the first six months of full population pilot testing in Missouri. J Pediatr 2015; 166:172-177.
45. van der Spek J, Groenwold RH, van der Burg M, et al. TREC based newborn screening for severe combined immunodeficiency disease: A systematic review. J Clin Immunol 2015;35:416-430.
46. Aldenhoven M, Wynn RF, Orchard PJ, et al. Longterm outcome of Hurler syndrome patients after hematopoietic cell transplantation: An international multicenter study. Blood 2015;125:2164-2172.
47. Frassoni F, Gualandi F, Podestà M, et al. Direct intrabone transplant of unrelated cord-blood cells in acute leukaemia: A Phase I/II study. Lancet Oncol 2008;9:831-839.
48. Saglio F, Berger M, Vassallo E, et al. Intrabone cord blood hematopoietic stem cell transplantation in a subset of very high-risk pediatric patients: A safety and feasibility pilot study. J Pediatr Hematol Oncol 2012;34:359-363.