115
Views
2
CrossRef citations to date
0
Altmetric
Key Paper Evaluations

A possible turning point in the hematopoietic stem cell gene therapy for primary immunodeficiency diseases? Lentiviral vectors could take the place of retroviral vectors

Pages 1015-1018 | Published online: 10 Jan 2014

References

  • Massaad MJ, Ramesh N, Geha RF. Wiskott-Aldrich syndrome: a comprehensive review. Ann. N.Y. Acad. Sci. 1285, 26–43 (2013).
  • Ariga T. Wiskott-Aldrich syndrome; an x-linked primary immunodeficiency disease with unique and characteristic features. Allergol. Int. 61(2), 183–189 (2012).
  • Derry JM, Ochs HD, Francke U. Isolation of a novel gene mutated in Wiskott-Aldrich syndrome. Cell 78, 635–644 (1994).
  • Ochs HD, Notarangelo LD. Structure and function of the Wiskott-Aldrich syndrome protein. Curr. Opin. Hematol. 12(4), 284–291 (2005).
  • Ariga T, Oda N, Yamaguchi K et al. T cell lines from two patients with adenosine deaminase (ADA) deficiency showed the restoration of ADA activity resulted from the reversion of an inherited mutation. Blood 97, 2896–2899 (2001).
  • Ariga T, Kondoh T, Yamaguchi K et al. Spontaneous in vivo reversion of an inherited mutation in the Wiskott-Aldrich syndrome. J. Immunol. 166, 5245–5249 (2001).
  • Seymour LW, Thrasher AJ. Gene therapy matures in the clinic. Nat. Biotechnol. 30(7), 588–593 (2012).
  • Boztug, K, Schmidt M, Schwarzer A et al. Stem-cell gene therapy for the Wiskott-Aldrich syndrome. N. Engl. J. Med. 360(20), 1918–1927 (2010).
  • Cavazzana-Calvo M, Fischer A, Hacein-Bey-Abina S et al. Gene therapy for primary immunodeficiencies: part 1. Curr. Opin. Immunol. 24, 580–584 (2012).
  • Hacein-Bey-Abina S, Garrigue A, Wang GP et al. Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J. Clin. Invest. 118, 3132–3142 (2008).
  • 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. 16, 198–204 (2010).
  • Aiuti A, Bacchetta R, Seger R et al. Gene therapy for primary immunodeficiencies: Part 2. Curr. Opin. Immunol. 24, 585–591(2012).
  • Scaramuzza S, Biasco L, Ripamonti A et al. Preclinical safety and efficacy of human CD34(+) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndrome. Mol. Ther. 21, 175–184 (2013).
  • Cartier N, Hacein-Bey-Abina S, Bartholomae CC et al. Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 326, 818–823 (2009).
  • Cavazzana-Calvo M, Payen E, Negre O et al. Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia. Nature 467, 318–322 (2010).
  • Aiuti A, Biasco L, Scaramuzza S et al. Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science 341(6148), 1233151 (2013).
  • Biffi A, Montini E, Lorioli L et al. Therapeutic benefit in metachromatic leukodystrophy by lentiviral hematopoietic stem cell gene therapy. Science 341(6148), 1233158 (2013).

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.