Gene therapy for SCID-X1: Focus on clinical data

Molecular Therapy

Volumn 19, Issue 12, 2011, Pages 2103-2104

  Baum, Christopher ^a  

^a NONE

Author keywords

[No Author keywords available]

Indexed keywords

HLA ANTIGEN; INTERLEUKIN 2 RECEPTOR GAMMA; RETROVIRUS VECTOR;

AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION; B LYMPHOCYTE; CLINICAL DECISION MAKING; DRUG EFFICACY; DRUG SAFETY; EDITORIAL; GENE THERAPY; GENOTOXICITY; HLA MATCHING; HUMAN; NATURAL KILLER CELL; RISK BENEFIT ANALYSIS; RISK REDUCTION; T LYMPHOCYTE; X LINKED SEVERE COMBINED IMMUNODEFICIENCY;

EID: 82955222192     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2011.252     Document Type: Editorial

References (14)

1. Gaspar, HB, Cooray, S, Gilmour, KC, Parsley, KL, Adams, S, Howe, SJ et al. (2011). Long-term persistence of a polyclonal T cell repertoire after gene therapy for X-linked severe combined immunodeficiency. Sci Transl Med 3:97ra79.
2. Hacein-Bey-Abina, S, Hauer, J, Lim, A, Picard, C, Wang, GP, Berry, CC et al. (2010). Efficacy of gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 363:355-364.
3. Antoine, C, Muller, S, Cant, A, Cavazzana-Calvo, M, Veys, P, Vossen, J et al. (2003). Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968-99. Lancet 361:553-560. (Pubitemid 36207472)
4. Gennery, AR, Slatter, MA, Grandin, L, Taupin, P, Cant, AJ, Veys, P 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.
5. Howe, SJ, Mansour, MR, Schwarzwaelder, K, Bartholomae, C, Hubank, M, Kempski, H 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.
6. Hacein-Bey-Abina, S, Garrigue, A, Wang, GP, Soulier, J, Lim, A, Morillon, E et al. (2008). Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J Clin Invest 118:3132-3142.
7. Thornhill, SI, Schambach, A, Howe, SJ, Ulaganathan, M, Grassman, E, Williams, D et al. (2008). Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency. Mol Ther 16:590-598. (Pubitemid 351315000)
8. Williams, DA, Thrasher, A and Baum, C (2010). Transatlantic consortium spotlights need for changes in gene therapy trials. Mol Ther 18:1892.
9. Cavazzana-Calvo, M, Fischer, A, Bushman, FD, Payen, E, Hacein-Bey-Abina, S and Leboulch, P (2011). Is normal hematopoiesis maintained solely by long-term multipotent stem cells? Blood 117:4420-4424.
10. Cavazzana-Calvo, M, Payen, E, Negre, O, Wang, G, Hehir, K, Fusil, F et al. (2010). Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia. Nature 467:318-322.
11. Stein, S, Ott, MG, Schultze-Strasser, S, Jauch, A, Burwinkel, B, Kinner, A et al. (2010). Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease. Nat Med 16:198-204.
12. Modlich, U, Navarro, S, Zychlinski, D, Maetzig, T, Knoess, S, Brugman, MH et al. (2009). Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors. Mol Ther 17:1919-1928.
13. Montini, E, Cesana, D, Schmidt, M, Sanvito, F, Bartholomae, CC, Ranzani, M et al. (2009). The genotoxic 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.
14. Cattoglio, C, Pellin, D, Rizzi, E, Maruggi, G, Corti, G, Miselli, F et al. (2010). Highdefinition mapping of retroviral integration sites identifies active regulatory elements in human multipotent hematopoietic progenitors. Blood 116:5507-5517.