Gene therapy for Wiskott–Aldrich syndrome

Bibliography

• Kirchhausen T, Rosen FS. Disease mechanism: unravelling Wiskott-Aldrich syndrome. Curr Biol CB 1996;6(6):676-8
   PubMed Web of Science ®Google Scholar
• Kiselar JG, Mahaffy R, Pollard TD, et al. Visualizing Arp2/3 complex activation mediated by binding of ATP and WASp using structural mass spectrometry. Proc Natl Acad Sci USA 2007;104(5):1552-7
   PubMed Web of Science ®Google Scholar
• Ochs HD. Mutations of the Wiskott-Aldrich Syndrome Protein affect protein expression and dictate the clinical phenotypes. Immunol Res 2009;44(1-3):84-8
   PubMed Web of Science ®Google Scholar
• Albert MH, Notarangelo LD, Ochs HD. Clinical spectrum, pathophysiology and treatment of the Wiskott-Aldrich syndrome. Curr Opin Hematol 2010; [Epub ahead of print]
   Web of Science ®Google Scholar
• Sullivan KE, Mullen CA, Blaese RM, Winkelstein JA. A multiinstitutional survey of the Wiskott-Aldrich syndrome. J Pediatrics 1994;125(6 Pt 1):876-85
   PubMed Web of Science ®Google Scholar
• Dupuis-Girod S, Medioni J, Haddad E, et al. Autoimmunity in Wiskott-Aldrich syndrome: risk factors, clinical features, and outcome in a single-center cohort of 55 patients. Pediatrics 2003;111(5 Pt 1):e622-7
   PubMed Web of Science ®Google Scholar
• Aldrich RA, Steinberg AG, Campbell DC. Pedigree demonstrating a sex-linked recessive condition characterized by draining ears, eczematoid dermatitis and bloody diarrhea. Pediatrics 1954;13(2):133-9
   PubMed Web of Science ®Google Scholar
• Derry JM, Ochs HD, Francke U. Isolation of a novel gene mutated in Wiskott-Aldrich syndrome. Cell 1994;78(4):635-44
   PubMed Web of Science ®Google Scholar
• Filipovich AH, Stone JV, Tomany SC, et al. Impact of donor type on outcome of bone marrow transplantation for Wiskott-Aldrich syndrome: collaborative study of the International Bone Marrow Transplant Registry and the National Marrow Donor Program. Blood 2001;97(6):1598-603
   PubMed Web of Science ®Google Scholar
• Moratto D, Giliani S, Bonfim C, et al. 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 2011;118(6):1675-84
   PubMed Web of Science ®Google Scholar
• Boztug K, Schmidt M, Schwarzer A, et al. Stem-cell gene therapy for the Wiskott-Aldrich syndrome. N Eng J med 2010;363(20):1918-27
   PubMed Web of Science ®Google Scholar
• Available from: www.ClinicalTrials.gov 2013 [Cited 25 July 2013]
   Google Scholar
• Blaese RM, Culver KW, Miller AD, et al. T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years. Science 1995;270(5235):475-80
   PubMed Web of Science ®Google Scholar
• Onodera M, Ariga T, Kawamura N, et al. Successful peripheral T-lymphocyte-directed gene transfer for a patient with severe combined immune deficiency caused by adenosine deaminase deficiency. Blood 1998;91(1):30-6
   PubMed Web of Science ®Google Scholar
• Onodera M, Nelson DM, Sakiyama Y, et al. Gene therapy for severe combined immunodeficiency caused by adenosine deaminase deficiency: improved retroviral vectors for clinical trials. Acta haematol 1999;101(2):89-96
   PubMed Web of Science ®Google Scholar
• Aiuti A, Slavin S, Aker M, et al. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 2002;296(5577):2410-13
   PubMed Web of Science ®Google Scholar
• 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(18):3635-46
   PubMed Web of Science ®Google Scholar
• Gaspar HB. Gene therapy for ADA-SCID: defining the factors for successful outcome. Blood 2012;120(18):3628-9
   PubMed Web of Science ®Google Scholar
• Aiuti A, Bacchetta R, Seger R, et al. Gene therapy for primary immunodeficiencies: Part 2. Curr Opin Immunol 2012;24(5):585-91
   PubMedGoogle Scholar
• Cassani B, Montini E, Maruggi G, et al. Integration of retroviral vectors induces minor changes in the transcriptional activity of T cells from ADA-SCID patients treated with gene therapy. Blood 2009;114(17):3546-56
   PubMed Web of Science ®Google Scholar
• Howe SJ, Mansour MR, Schwarzwaelder K, et al. Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. J Clin Invest 2008;118(9):3143-50
   PubMed Web of Science ®Google Scholar
• 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(97):97ra79
   PubMed Web of Science ®Google Scholar
• Hacein-Bey-Abina S, Hauer J, Lim A, et al. Efficacy of gene therapy for X-linked severe combined immunodeficiency. N Eng J Med 2010;363(4):355-64
   PubMed Web of Science ®Google Scholar
• Baum C. Gene therapy for SCID-X1: focus on clinical data. Mol Ther 2011;19(12):2103-4
   PubMed Web of Science ®Google Scholar
• Grez M, Reichenbach J, Schwable J, et al. Gene therapy of chronic granulomatous disease: the engraftment dilemma. Mol Ther 2011;19(1):28-35
   PubMed Web of Science ®Google Scholar
• Ott MG, Schmidt M, Schwarzwaelder K, et al. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat Med 2006;12(4):401-9
   PubMed Web of Science ®Google Scholar
• European Society of Gene Therapy. One of three successfully treated CGD patients in a Swiss-German gene therapy trial died due to his underlying disease: a position statement from the European Society of Gene Therapy (ESGT). J Gene Med 2006;8(12):1435
   PubMed Web of Science ®Google Scholar
• Kang HJ, Bartholomae CC, Paruzynski A, et al. Retroviral gene therapy for X-linked chronic granulomatous disease: results from phase I/II trial. Mol Ther 2011;19(11):2092-101
   PubMed Web of Science ®Google Scholar
• Stein S, Ott MG, Schultze-Strasser S, et al. Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease. Nat Med 2010;16(2):198-204
   PubMed Web of Science ®Google Scholar
• Klein C, Nguyen D, Liu CH, et al. Gene therapy for Wiskott-Aldrich syndrome: rescue of T-cell signaling and amelioration of colitis upon transplantation of retrovirally transduced hematopoietic stem cells in mice. Blood 2003;101(6):2159-66
   PubMed Web of Science ®Google Scholar
• Snapper SB, Meelu P, Nguyen D, et al. WASP deficiency leads to global defects of directed leukocyte migration in vitro and in vivo. J Leukoc Biol 2005;77(6):993-8
   PubMed Web of Science ®Google Scholar
• Dewey RA, Avedillo Diez I, Ballmaier M, et al. Retroviral WASP gene transfer into human hematopoietic stem cells reconstitutes the actin cytoskeleton in myeloid progeny cells differentiated in vitro. Exp Hematol 2006;34(9):1161-9
   PubMed Web of Science ®Google Scholar
• Blundell MP, Bouma G, Calle Y, et al. Improvement of migratory defects in a murine model of Wiskott-Aldrich syndrome gene therapy. Mol Ther 2008;16(5):836-44
   PubMed Web of Science ®Google Scholar
• Boztug K, Dewey RA, Klein C. Development of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome. Curr Opin Mol Ther 2006;8(5):390-5
   PubMedGoogle Scholar
• Schilz AJ, Kuhlcke K, Fauser AA, Eckert HG. Optimization of retroviral vector generation for clinical application. J Gene Med 2001;3(5):427-36
   PubMed Web of Science ®Google Scholar
• Modlich U, Baum C. Preventing and exploiting the oncogenic potential of integrating gene vectors. J Clin Invest 2009;119(4):755-8
   PubMed Web of Science ®Google Scholar
• Maruggi G, Porcellini S, Facchini G, et al. Transcriptional enhancers induce insertional gene deregulation independently from the vector type and design. Mol Ther 2009;17(5):851-6
   PubMed Web of Science ®Google Scholar
• Astrakhan A, Sather BD, Ryu BY, et al. Ubiquitous high-level gene expression in hematopoietic lineages provides effective lentiviral gene therapy of murine Wiskott-Aldrich syndrome. Blood 2012;119(19):4395-407
   PubMed Web of Science ®Google Scholar
• Dupre L, Trifari S, Follenzi A, et al. Lentiviral vector-mediated gene transfer in T cells from Wiskott-Aldrich syndrome patients leads to functional correction. Mol Ther 2004;10(5):903-15
   PubMed Web of Science ®Google Scholar
• Charrier S, Dupre L, Scaramuzza S, et al. Lentiviral vectors targeting WASp expression to hematopoietic cells, efficiently transduce and correct cells from WAS patients. Gene Ther 2007;14(5):415-28
   PubMed Web of Science ®Google Scholar
• Papayannakos C, Daniel R. Understanding lentiviral vector chromatin targeting: working to reduce insertional mutagenic potential for gene therapy. Gene Ther 2013;20(6):581-8
   PubMed Web of Science ®Google Scholar
• Thornhill SI, Schambach A, Howe SJ, et al. Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency. Mol Ther 2008;16(3):590-8
   PubMed Web of Science ®Google Scholar
• Avedillo Diez I, Zychlinski D, Coci EG, et al. Development of novel efficient SIN vectors with improved safety features for Wiskott-Aldrich syndrome stem cell based gene therapy. Mol Pharm 2011;8(5):1525-37
   PubMed Web of Science ®Google Scholar
• Scaramuzza S, Biasco L, Ripamonti A, et al. Preclinical Safety and Efficacy of Human CD 34+ Cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich Syndrome. Mol Ther 2013;21(1):175-84
   PubMed Web of Science ®Google Scholar
• Galy A, Thrasher AJ. Gene therapy for the Wiskott-Aldrich syndrome. Curr Opin Allergy Clin Immunol 2011;11(6):545-50
   PubMed Web of Science ®Google Scholar
• Aiuti A, Biasco L, Scaramuzza S, et al. Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome. Science 2013; [Epub ahead of print]
   Google Scholar
• Knight S, Collins M, Takeuchi Y. Insertional mutagenesis by retroviral vectors: current concepts and methods of analysis. Curr Gene Ther 2013;13(3):211-27
   PubMed Web of Science ®Google Scholar
• Nakamura T. Retroviral insertional mutagenesis identifies oncogene cooperation. Cancer Sci 2005;96(1):7-12
   PubMed Web of Science ®Google Scholar
• Trobridge GD. Genotoxicity of retroviral hematopoietic stem cell gene therapy. Expert Opin Biol Ther 2011;11(5):581-93
   PubMed Web of Science ®Google Scholar
• Biffi A, Montini E, Lorioli L, et al. Lentiviral Hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science 2013; [Epub ahead of print]
   Web of Science ®Google Scholar
• 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(5954):818-23
   PubMed Web of Science ®Google Scholar
• Carson DA, Wasson DB, Beutler E. Antileukemic and immunosuppressive activity of 2-chloro-2′-deoxyadenosine. Proc Natl Acad Sci USA 1984;81(7):2232-6
   PubMed Web of Science ®Google Scholar
• Baum C, Modlich U, Gohring G, Schlegelberger B. Concise review: managing genotoxicity in the therapeutic modification of stem cells. Stem Cells 2011;29(10):1479-84
   PubMed Web of Science ®Google Scholar
• Sellers S, Gomes TJ, Larochelle A, et al. Ex vivo expansion of retrovirally transduced primate CD 34+ cells results in overrepresentation of clones with MDS1/EVI1 insertion sites in the myeloid lineage after transplantation. Mol Ther 2010;18(9):1633-9
   PubMed Web of Science ®Google Scholar
• Curtis DJ, McCormack MP. The molecular basis of Lmo2-induced T-cell acute lymphoblastic leukemia. Clin Cancer Res 2010;16(23):5618-23
   PubMed Web of Science ®Google Scholar
• Cleveland SM, Smith S, Tripathi R, et al. Lmo2 induces hematopoietic stem cell-like features in T-cell progenitor cells prior to leukemia. Stem Cells 2013;31(5):882-94
   PubMed Web of Science ®Google Scholar
• Tatarek J, Cullion K, Ashworth T, et al. Notch1 inhibition targets the leukemia-initiating cells in a Tal1/Lmo2 mouse model of T-ALL. Blood 2011;118(6):1579-90
   PubMed Web of Science ®Google Scholar
• Cavazzana-Calvo M, Payen E, Negre O, et al. Transfusion independence and HMGA2 activation after gene therapy of human beta-thalassaemia. Nature 2010;467(7313):318-22
   PubMed Web of Science ®Google Scholar
• Wefers B, Meyer M, Ortiz O, et al. Direct production of mouse disease models by embryo microinjection of TALENs and oligodeoxynucleotides. Proc Natl Acad Sci USA 2013;110(10):3782-7
   PubMed Web of Science ®Google Scholar
• Osborn MJ, Starker CG, McElroy AN, et al. TALEN-based gene correction for epidermolysis bullosa. Mol Ther 2013;21(6):1151-9
   PubMed Web of Science ®Google Scholar
• Kurosawa A, Saito S, Mori M, Adachi N. Nucleofection-based gene targeting in human pre-B cells. Gene 2012;492(1):305-8
   PubMed Web of Science ®Google Scholar
• Moore JC, Atze K, Yeung PL, et al. Efficient, high-throughput transfection of human embryonic stem cells. Stem Cell Res Ther 2010;1(3):23
   PubMedGoogle Scholar
• Nagaria P, Robert C, Rassool FV. DNA double-strand break response in stem cells: mechanisms to maintain genomic integrity. Biochim Biophys Acta 2013;1830(2):2345-53
   PubMed Web of Science ®Google Scholar
• Dahlem TJ, Hoshijima K, Jurynec MJ, et al. Simple methods for generating and detecting locus-specific mutations induced with TALENs in the zebrafish genome. PLoS Genet 2012;8(8):e1002861
   PubMed Web of Science ®Google Scholar
• Ding Q, Lee YK, Schaefer EA, et al. A TALEN genome-editing system for generating human stem cell-based disease models. Cell Stem Cell 2013;12(2):238-51
   PubMed Web of Science ®Google Scholar
• Mussolino C, Morbitzer R, Lutge F, et al. A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity. Nucleic acids Res 2011;39(21):9283-93
   PubMed Web of Science ®Google Scholar
• Friedrich W, Schutz C, Schulz A, et al. Results and long-term outcome in 39 patients with Wiskott-Aldrich syndrome transplanted from HLA-matched and -mismatched donors. Immunol Res 2009;44(1-3):18-24
   PubMed Web of Science ®Google Scholar
• Heinemann T, Heinrichs B, Klein C, et al. The “controlled individualized treatment” as a new instrument in first-time clinical appliacation of high-risk therapy forms- ethics analysis of somatic gene therapy for Wiskott-Aldrich Syndrome (Article in German). 11 edition. De Gryuter; Berlin: 2006
   Google Scholar