Corrigendum to “Preclinical Safety and Efficacy of Human CD34+ Cells Transduced With Lentiviral Vector for the Treatment of Wiskott-Aldrich Syndrome” (Molecular Therapy (2013) 21(1) (175–184)(S1525001616305731)(10.1038/mt.2012.23));Preclinical Safety and Efficacy of human CD34 + Cells transduced with lentiviral vector for the treatment of wiskott-aldrich syndrome

Molecular Therapy

Volumn 21, Issue 1, 2013, Pages 175-184

  Scaramuzza, Samantha ^a,b   Biasco, Luca ^a,c   Ripamonti, Anna ^a   Castiello, Maria C ^a   Loperfido, Mariana ^a   Draghici, Elena ^a   Hernandez, Raisa J ^a   Benedicenti, Fabrizio ^a   Radrizzani, Marina ^d   Salomoni, Monica ^d   Ranzani, Marco ^a,c   Bartholomae, Cynthia C ^e   Vicenzi, Elisa ^f   Finocchi, Andrea ^g,h   Bredius, Robbert ⁱ   Bosticardo, Marita ^a   Schmidt, Manfred ^e   Von Kalle, Christof ^e   Montini, Eugenio ^a   Biffi, Alessandra ^a,j   Roncarolo, Maria G ^a,c,j   Naldini, Luigi ^a,c   Villa, Anna ^a,b   Aiuti, Alessandro ^a,h   more..

^a SAN RAFFAELE SCIENTIFIC INSTITUTE   (Italy)

^b CNR   (Italy)

^c VITA SALUTE SAN RAFFAELE UNIVERSITY   (Italy)

^d MOLMED S P A   (Italy)

^e GERMAN CANCER RESEARCH CENTER DKFZ   (Germany)

^f San Raf faele Institute   (Italy)

^g BAMBINO GESÙ CHILDREN S HOSPITAL   (Italy)

^h UNIVERSITY OF ROME TOR VERGATA   (Italy)

ⁱ LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

^j SAN RAFFAELE HOSPITAL   (Italy)

more..

Author keywords

[No Author keywords available]

Indexed keywords

LENTIVIRUS VECTOR; PROTEIN WAS; UNCLASSIFIED DRUG; VIRUS PROTEIN;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BONE MARROW CELL; CD34 SELECTION; CELLULAR DISTRIBUTION; CLINICAL EFFECTIVENESS; CONTROLLED STUDY; GERM LINE GENE THERAPY; HUMAN; HUMAN CELL; IMMOBILIZED CELL; IN VITRO STUDY; IN VIVO GENE TRANSFER; IN VIVO STUDY; LYMPHOID CELL; MOLECULAR MODEL; MOUSE; NEWBORN; NONHUMAN; PROTEIN EXPRESSION; SIGNAL TRANSDUCTION; STEM CELL GENE THERAPY; VALIDATION PROCESS; VIRAL GENE DELIVERY SYSTEM; VIRUS CELL INTERACTION; VIRUS PURIFICATION; VIRUS SHEDDING; VIRUS TRANSMISSION; WISKOTT ALDRICH SYNDROME;

MUS;

EID: 84871929094     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2016.96     Document Type: Erratum

References (45)

1. Aiuti, A and Roncarolo, MG (2009). Ten years of gene therapy for primary immunedeficiencies. Hematology Am Soc Hematol Educ Program: 682-689.
2. Fischer, A, Hacein-Bey-Abina, S and Cavazzana-Calvo, M (2010). 20 years of genetherapy for SCID. Nat Immunol 11: 457-460.
3. Aiuti, A, Cattaneo, F, Galimberti, S, Benninghoff, U, Cassani, B, Callegaro, L et al.(2009). Gene therapy for immunodeficiency due to adenosine deaminase deficiency.N Engl J Med 360: 447-458.
4. 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 JMed 363: 355-364.
5. Gaspar, HB, Parsley, KL, Howe, S, King, D, Gilmour, KC, Sinclair, J et al. (2004). Genetherapy of X-linked severe combined immunodeficiency by use of a pseudotypedgammaretroviral vector. Lancet 364: 2181-2187. (Pubitemid 40018447)
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 ofSCID-X1. J Clin Invest 118: 3132-3142.
7. Howe, SJ, Mansour, MR, Schwarzwaelder, K, Bartholomae, C, Hubank, M, Kempski,H et al. (2008). Insertional mutagenesis combined with acquired somatic mutationscauses leukemogenesis following gene therapy of SCID-X1 patients. J Clin Invest 118:3143-3150.
8. Stein, S, Ott, MG, Schultze-Strasser, S, Jauch, A, Burwinkel, B, Kinner, A et al.(2010). Genomic instability and myelodysplasia with monosomy 7 consequent toEVI1 activation after gene therapy for chronic granulomatous disease. Nat Med 16:198-204.
9. Montini, E, Cesana, D, Schmidt, M, Sanvito, F, Ponzoni, M, Bartholomae, C et al.(2006). Hematopoietic stem cell gene transfer in a tumor-prone mouse modeluncovers low genotoxicity of lentiviral vector integration. Nat Biotechnol 24: 687-696. (Pubitemid 43882120)
10. Cartier, N, Hacein-Bey-Abina, S, Bartholomae, CC, Veres, G, Schmidt, M, Kutschera, Iet al. (2009). Hematopoietic stem cell gene therapy with a lentiviral vector in X-linkedadrenoleukodystrophy. Science 326: 818-823.
11. 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 humanß-thalassaemia. Nature 467: 318-322.
12. Filipovich, AH, Stone, JV, Tomany, SC, Ireland, M, Kollman, C, Pelz, CJ et al. (2001).Impact of donor type on outcome of bone marrow transplantation for Wiskott-Aldrichsyndrome: collaborative study of the International Bone Marrow Transplant Registryand the National Marrow Donor Program. Blood 97: 1598-1603. (Pubitemid 32217223)
13. Ozsahin, H, Cavazzana-Calvo, M, Notarangelo, LD, Schulz, A, Thrasher, AJ, Mazzolari,E et al. (2008). Long-term outcome following hematopoietic stem-cell transplantationin Wiskott-Aldrich syndrome: collaborative study of the European Society forImmunodeficiencies and European Group for Blood and Marrow Transplantation.Blood 111: 439-445.
14. Shcherbina, A, Candotti, F, Rosen, FS and Remold-O'Donnell, E (2003). Highincidence of lymphomas in a subgroup of Wiskott-Aldrich syndrome patients. Br JHaematol 121: 529-530. (Pubitemid 36628350)
15. Ochs, HD and Thrasher, AJ (2006). The Wiskott-Aldrich syndrome. J Allergy ClinImmunol 117: 725-38; quiz 739. (Pubitemid 44313817)
16. Derry, JM, Ochs, HD and Francke, U (1994). Isolation of a novel gene mutated inWiskott-Aldrich syndrome. Cell 79: following 922.
17. Ramesh, N and Geha, R (2009). Recent advances in the biology of WASP and WIP.Immunol Res 44: 99-111.
18. Bosticardo, M, Marangoni, F, Aiuti, A, Villa, A and Grazia Roncarolo, M (2009). Recentadvances in understanding the pathophysiology of Wiskott-Aldrich syndrome. Blood113: 6288-6295.
19. Boztug, K, Dewey, RA and Klein, C (2006). Development of hematopoietic stem cellgene therapy for Wiskott-Aldrich syndrome. Curr Opin Mol Ther 8: 390-395. (Pubitemid 44566780)
20. Boztug, K, Schmidt, M, Schwarzer, A, Banerjee, PP, Díez, IA, Dewey, RA et al.(2010). Stem-cell gene therapy for the Wiskott-Aldrich syndrome. N Engl J Med 363:1918-1927.
21. Avedillo Díez, I, Zychlinski, D, Coci, EG, Galla, M, Modlich, U, Dewey, RA 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.
22. Dupré, L, Marangoni, F, Scaramuzza, S, Trifari, S, Hernández, RJ, Aiuti, A et al. (2006).Efficacy of gene therapy for Wiskott-Aldrich syndrome using a WAS promoter/cDNAcontaininglentiviral vector and nonlethal irradiation. Hum Gene Ther 17: 303-313.
23. Marangoni, F, Bosticardo, M, Charrier, S, Draghici, E, Locci, M, Scaramuzza, S et al.(2009). Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrichsyndrome in preclinical models. Mol Ther 17: 1073-1082.
24. Dupré, L, Trifari, S, Follenzi, A, Marangoni, F, Lain de Lera, T, Bernad, A et al. (2004).Lentiviral vector-mediated gene transfer in T cells from Wiskott-Aldrich syndromepatients leads to functional correction. Mol Ther 10: 903-915. (Pubitemid 39419917)
25. Charrier, S, Dupré, L, Scaramuzza, S, Jeanson-Leh, L, Blundell, MP, Danos, O et al.(2007). Lentiviral vectors targeting WASp expression to hematopoietic cells, efficientlytransduce and correct cells from WAS patients. Gene Ther 14: 415-428.
26. .
27. Traggiai, E, Chicha, L, Mazzucchelli, L, Bronz, L, Piffaretti, JC, Lanzavecchia, A et al.(2004). Development of a human adaptive immune system in cord blood celltransplantedmice. Science 304: 104-107. (Pubitemid 38451466)
28. Benhamida, S, Pflumio, F, Dubart-Kupperschmitt, A, Zhao-Emonet, JC,Cavazzana-Calvo, M, Rocchiccioli, F et al. (2003). Transduced CD34+ cells fromadrenoleukodystrophy patients with HIV-derived vector mediate long-termengraftment of NOD/SCID mice. Mol Ther 7: 317-324. (Pubitemid 36503855)
29. Ficara, F, Superchi, DB, Hernández, RJ, Mocchetti, C, Carballido-Perrig, N, Andolfi, Get al. (2004). IL-3 or IL-7 increases ex vivo gene transfer efficiency in ADA-SCID BMCD34+ cells while maintaining in vivo lymphoid potential. Mol Ther 10: 1096-1108. (Pubitemid 39625253)
30. Cornils, K, Lange, C, Schambach, A, Brugman, MH, Nowak, R, Lioznov, M et al.(2009). Stem cell marking with promotor-deprived self-inactivating retroviral vectorsdoes not lead to induced clonal imbalance. Mol Ther 17: 131-143.
31. Will, E, Bailey, J, Schuesler, T, Modlich, U, Balcik, B, Burzynski, B et al. (2007).Importance of murine study design for testing toxicity of retroviral vectors in supportof phase I trials. Mol Ther 15: 782-791. (Pubitemid 46431999)
32. Pan, YW, Scarlett, JM, Luoh, TT and Kurre, P (2007). Prolonged adherence of humanimmunodeficiency virus-derived vector particles to hematopoietic target cells leads tosecondary transduction in vitro and in vivo. J Virol 81: 639-649. (Pubitemid 46067891)
33. Mani, M, Venkatasubrahmanyam, S, Sanyal, M, Levy, S, Butte, A, Weinberg, K et al.(2009). Wiskott-Aldrich syndrome protein is an effector of Kit signaling. Blood 114:2900-2908.
34. Ishikawa, F, Yasukawa, M, Lyons, B, Yoshida, S, Miyamoto, T, Yoshimoto, G et al.(2005). Development of functional human blood and immune systems in NOD/SCID/IL2 receptor {gamma} chain(null) mice. Blood 106: 1565-1573. (Pubitemid 41208567)
35. Follenzi, A, Sabatino, G, Lombardo, A, Boccaccio, C and Naldini, L (2002). Efficientgene delivery and targeted expression to hepatocytes in vivo by improved lentiviralvectors. Hum Gene Ther 13: 243-260. (Pubitemid 34164109)
36. Cattoglio, C, Facchini, G, Sartori, D, Antonelli, A, Miccio, A, Cassani, B et al. (2007).Hot spots of retroviral integration in human CD34+ hematopoietic cells. Blood 110:1770-1778. (Pubitemid 47443887)
37. Mantovani, J, Charrier, S, Eckenberg, R, Saurin, W, Danos, O, Perea, J et al. (2009).Diverse genomic integration of a lentiviral vector developed for the treatment ofWiskott-Aldrich syndrome. J Gene Med 11: 645-654.
38. Modlich, U, Navarro, S, Zychlinski, D, Maetzig, T, Knoess, S, Brugman, MH et al.(2009). Insertional transformation of hematopoietic cells by self-inactivating lentiviraland gammaretroviral vectors. Mol Ther 17: 1919-1928.
39. Galy, A, Roncarolo, MG and Thrasher, AJ (2008). Development of lentiviral genetherapy for Wiskott Aldrich syndrome. Expert Opin Biol Ther 8: 181-190. (Pubitemid 351233711)
40. Galy, A and Thrasher, AJ (2011). Gene therapy for the Wiskott-Aldrich syndrome. CurrOpin Allergy Clin Immunol 11: 545-550.
41. Zhu, Q, Watanabe, C, Liu, T, Hollenbaugh, D, Blaese, RM, Kanner, SB et al. (1997).Wiskott-Aldrich syndrome/X-linked thrombocytopenia: WASP gene mutations, proteinexpression, and phenotype. Blood 90: 2680-2689. (Pubitemid 27413478)
42. Trifari, S, Scaramuzza, S, Catucci, M, Ponzoni, M, Mollica, L, Chiesa, R et al. (2010).Revertant T lymphocytes in a patient with Wiskott-Aldrich syndrome: analysis offunction and distribution in lymphoid organs. J Allergy Clin Immunol 125: 439-448.e8.
43. Farson, D, Witt, R, McGuinness, R, Dull, T, Kelly, M, Song, J et al. (2001). A newgenerationstable inducible packaging cell line for lentiviral vectors. Hum Gene Ther12: 981-997. (Pubitemid 32681861)
44. Escarpe, P, Zayek, N, Chin, P, Borellini, F, Zufferey, R, Veres, G et al. (2003).Development of a sensitive assay for detection of replication-competent recombinantlentivirus in large-scale HIV-based vector preparations. Mol Ther 8: 332-341. (Pubitemid 37062843)
45. Schmidt, M, Schwarzwaelder, K, Bartholomae, C, Zaoui, K, Ball, C, Pilz, I et al. (2007).High-resolution insertion-site analysis by linear amplification-mediated PCR (LAMPCR).Nat Methods 4: 1051-1057. (Pubitemid 350201778)