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-Reference-Cited by-同舟云学术

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

Published:2019-05 Issue:5 Volume:6 Page:e239-e253
ISSN:2352-3026
Container-title:The Lancet Haematology
language:en
Short-container-title:The Lancet Haematology

Author:

Ferrua Francesca,Cicalese Maria Pia,Galimberti Stefania,Giannelli Stefania,Dionisio Francesca,Barzaghi Federica,Migliavacca Maddalena,Bernardo Maria Ester,Calbi Valeria,Assanelli Andrea Angelo,Facchini Marcella,Fossati Claudia,Albertazzi Elena,Scaramuzza Samantha,Brigida Immacolata,Scala Serena,Basso-Ricci Luca,Pajno Roberta,Casiraghi Miriam,Canarutto Daniele,Salerio Federica Andrea,Albert Michael H,Bartoli Antonella,Wolf Hermann M,Fiori Rossana,Silvani Paolo,Gattillo Salvatore,Villa Anna,Biasco Luca,Dott Christopher,Culme-Seymour Emily J,van Rossem Koenraad,Atkinson Gillian,Valsecchi Maria Grazia,Roncarolo Maria Grazia,Ciceri Fabio,Naldini Luigi,Aiuti Alessandro

Publisher

Elsevier BV

Subject

Hematology

Reference33 articles.

1. WASP: a key immunological multitasker;Thrasher;Nat Rev Immunol,2010

2. The Wiskott-Aldrich syndrome;Ochs;J Allergy Clin Immunol,2006

3. Recent advances in understanding the pathophysiology of Wiskott-Aldrich syndrome;Bosticardo;Blood,2009

4. A multi-institutional survey of the Wiskott-Aldrich syndrome;Sullivan;J Pediatr,1994

5. Autoimmunity in Wiskott-Aldrich syndrome: risk factors, clinical features, and outcome in a single-center cohort of 55 patients;Dupuis-Girod;Pediatrics,2003

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