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5. Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study. Ferrua F; Cicalese MP; Galimberti S; Giannelli S; Dionisio F; Barzaghi F; Migliavacca M; Bernardo ME; Calbi V; Assanelli AA; Facchini M; Fossati C; Albertazzi E; Scaramuzza S; Brigida I; Scala S; Basso-Ricci L; Pajno R; Casiraghi M; Canarutto D; Salerio FA; Albert MH; Bartoli A; Wolf HM; Fiori R; Silvani P; Gattillo S; Villa A; Biasco L; Dott C; Culme-Seymour EJ; van Rossem K; Atkinson G; Valsecchi MG; Roncarolo MG; Ciceri F; Naldini L; Aiuti A Lancet Haematol; 2019 May; 6(5):e239-e253. PubMed ID: 30981783 [TBL] [Abstract][Full Text] [Related]
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13. [Analysis of clinical features and gene mutations in 6 patients with Wiskott-Aldrich syndrome]. Jiang MH; Wang ZY; Su J; Cao LJ; Li JQ; Sun XH; Bai X; Wang GF; Ruan CG Zhonghua Xue Ye Xue Za Zhi; 2011 Sep; 32(9):577-82. PubMed ID: 22338148 [TBL] [Abstract][Full Text] [Related]
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