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Miriam Casiraghi
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Journal Articles

Gene Therapy for Beta Thalassemia: Preliminary Results from the PHASE I/II Tiget-Bthal Trial of Autologous Hematopoietic Stem Cells Genetically Modified with GLOBE Lentiviral Vector

Sarah Marktel, MD, Maria Pia Cicalese, Fabio Giglio, MD, Samantha Scaramuzza, Valeria Calbi, Miriam Casiraghi, Francesca Ciotti, Maria Rosa Lidonnici, Claudia Rossi, Nicoletta Masera, Emanuela D'Angelo, Nadia Mirra, Raffaella Origa, MD, Immacolata Tartaglione, Giacomo Mandelli, Raffaella Milani, Salvatore Gattillo, Milena Coppola, Gianluca Viarengo, Luca Santoleri, Andrea Calabria, Silverio Perrotta, Eugenio Montini, Giovanna Graziadei, MD, Luigi Naldini, MD PhD, Maria Domenica Cappellini, MD, Fabio Ciceri, MD, Alessandro Aiuti, Giuliana Ferrari
Journal: Blood
Blood (2017) 130 (Supplement 1): 355.
https://doi.org/10.1182/blood.V130.Suppl_1.355.355
Published: 2017
Journal Articles

Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency

Maria Pia Cicalese, Francesca Ferrua, Laura Castagnaro, Roberta Pajno, Federica Barzaghi, Stefania Giannelli, Francesca Dionisio, Immacolata Brigida, Marco Bonopane, Miriam Casiraghi, Antonella Tabucchi, Filippo Carlucci, Eyal Grunebaum, Mehdi Adeli, Robbert G. Bredius, Jennifer M. Puck, Polina Stepensky, Ilhan Tezcan, Katie Rolfe, Erika De Boever, Rickey R. Reinhardt, Jonathan Appleby, Fabio Ciceri, Maria Grazia Roncarolo, Alessandro Aiuti
Journal: Blood
Blood (2016) 128 (1): 45–54.
https://doi.org/10.1182/blood-2016-01-688226
Published: 2016
Includes: Supplemental data
Journal Articles

Safety and Clinical Benefit of Lentiviral Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome

Francesca Ferrua, MD, Maria Pia Cicalese, MD PhD, Stefania Galimberti, PhD, Samantha Scaramuzza, PhD, Stefania Giannelli, PhD, Roberta Pajno, MD, Francesca Dionisio, PhD, Luca Biasco, PhD, Maria Carmina Castiello, PhD, Miriam Casiraghi, RN, Marcella Facchini, PhD, Andrea Finocchi, MD, Ayse Metin, MD PhD, Jordan S. Orange, MD PhD, Michael H. Albert, MD, Carmen Petrescu, MD, Marita Bosticardo, PhD, Anna Villa, MD, Chris Dott, PhD, Koen van Rossem, MD MSc, PhD, Maria Grazia Valsecchi, PhD, Fabio Ciceri, MD, Maria Grazia Roncarolo, MD, Luigi Naldini, MD PhD, Alessandro Aiuti, MD PhD
Journal: Blood
Blood (2015) 126 (23): 259.
https://doi.org/10.1182/blood.V126.23.259.259
Published: 2015
Journal Articles

ADA-deficient SCID is associated with a specific microenvironment and bone phenotype characterized by RANKL/OPG imbalance and osteoblast insufficiency

Aisha V. Sauer, Emanuela Mrak, Raisa Jofra Hernandez, Elena Zacchi, Francesco Cavani, Miriam Casiraghi, Eyal Grunebaum, Chaim M. Roifman, Maria C. Cervi, Alessandro Ambrosi, Filippo Carlucci, Maria Grazia Roncarolo, Anna Villa, Alessandro Rubinacci, Alessandro Aiuti
Journal: Blood
Blood (2009) 114 (15): 3216–3226.
https://doi.org/10.1182/blood-2009-03-209221
Published: 2009
Includes: Supplemental data