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Roberto Gambari, PhD
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Journal Articles

Breakthroughs in Preclinical Development of Ataluren (PTC124) As Therapeutic Option for Patients Affected By Shwachman-Diamond Syndrome: Towards the First Clinical Trial

Valentino Bezzerri, PhD, Antonio Vella, PhD, Elisabetta D'Aversa, PhD, Martina Api, PhD, Marisole Allegri, MSc, Elena Marinelli Busilacchi, PhD, Giovanna D'Amico, BSc, PhD MSc, Roberto Gambari, PhD, Claudio Sorio, MD PhD, Benedetta Fabrizzi, MD, Vincenzo Bronte, MD, Antonella Poloni, PhD MD, Monica Borgatti, PhD, Marco Cipolli, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 451.
https://doi.org/10.1182/blood-2019-127866
Published: 2019
Journal Articles

A Signature of Differentially Expressed Micrornas in Lymphoblastoid Cells from Shwachman-Diamond Syndrome Patients Indicates Possible Molecular Targets for Mirna Therapeutics

Jessica Gasparello, PhD, Chiara Papi, Giulia Breveglieri, PhD, Elisabetta D'Aversa, PhD, Valentino Bezzerri, PhD, Giovanna D'Amico, BSc, PhD MSc, Marco Cipolli, MD, Roberto Gambari, PhD, Monica Borgatti, PhD, Alessia Finotti, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2504.
https://doi.org/10.1182/blood-2019-128607
Published: 2019
Journal Articles

Following Beta-Globin Gene Transfer, the Production of Hemoglobin Depends Upon the Beta-Thalassemia Genotype.

Laura Breda, PhD, Carla Casu, PhD, Laura Casula, PhD, Dorothy A. Kleinert, NP, MPH, Nicoletta Bianchi, PhD, Evgenia Prus, PhD, Luca Cartegni, PhD, Eitan Fibach, PhD, Lawrence B. Gardner, MD, Patricia J Giardina, MD, Roberto Gambari, PhD, Stefano Rivella, PhD
Journal: Blood
Blood (2009) 114 (22): 978.
https://doi.org/10.1182/blood.V114.22.978.978
Published: 2009