|
1）Gaspar HB, Aiuti A, Porta F, et al. How I treat ADA deficiency. Blood. 2009; 114: 3524-32
|
|
|
|
2）Hassan A, Booth C, Brightwell A, et al. Outcome of hematopoietic stem cell transplantation for adenosine deaminase-deficient severe combined immunodeficiency. Blood. 2012; 120: 3615-24
|
|
|
|
|
|
3）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: 475-80
|
|
|
|
|
|
4）Aiuti A, Cattaneo F, Galimberti S, et al. Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N Engl J Med. 2009; 360: 447-58
|
|
|
|
|
|
5）Antoine C, Müller S, Cant A, et al. Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968-99. Lancet. 2003; 361: 553-60
|
|
|
|
|
|
6）Grunebaum E, Mazzolari E, Porta F,et al. Bone marrow transplantation for severe combined immune deficiency. JAMA. 2006; 295: 508-18
|
|
|
|
|
|
7）Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science. 2000; 288: 669-72
|
|
|
|
|
|
8）Hacein-Bey-Abina S, Hauer J, Lim A, et al. Efficacy of gene therapy for X-linked severe combined immunodeificiency. N Engl J Med. 2010; 363: 355-64
|
|
|
|
|
|
9）Kobayashi R, Ariga T, Nonoyama S, et al. Outcome in patients with Wiskott-Aldrich syndrome following stem cell transplantation: an analysis of 57 patients in Japan. Br J Haematol. 2006; 135: 362-6
|
|
|
|
|
|
10）Ozsahin H, Cavazzana-Calvo M, Notarangelo LD, et al. Long-term outcome following hematopoietic stem-cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and European Group for Blood and Marrow Transplantation. Blood. 2008; 111: 439-45
|
|
|
|
|
|
11）Boztug K, Schmidt M, Schwarzer A, et al. Stem-cell gene therapy for the Wiskott-Aldrich syndrome. N Engl J Med. 2010; 363: 1918-27
|
|
|
|
|
|
12）Braun CJ, Boztug K, Paruzynski A, et al. Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity. Sci Transl Med. 2014; 6: 227
|
|
|
|
|
|
13）Aiuti A, Biasco L, Scaramuzza S, et al. Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science. 2013; 34: 1233151
|
|
|
|
|
|
14）Kang EM, Choi U, Theobald N, et al. Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long term-correction of oxidase activity in peripheral blood neutrophils. Blood. 2009; 115: 783-91
|
|
|
|
|
|
15）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: 198-204
|
|
|
|
|
|
16）Griffith LM, Cowan MJ, Notarangelo LD, et al. Primary Immune Deficiency Treatment Consortium (PIDTC) report. Allergy Clin Immunol. 2014; 133: 335-47
|
|
|
|
|