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
Titel:
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
Auteur:
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
Verschenen in:
The Lancet. Haematology
Paginering:
Jaargang 6 (2019) nr. 5 pagina's e239-e253
Jaar:
2019
Inhoud:
Uitgever:
The Author(s). Published by Elsevier Ltd. This is an Open Access article under the CC BY 4.0 license