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Development of gene therapy and genome editing strategies to treat adenosine deaminase 2 deficiency

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Publications

Lentiviral correction of enzymatic activity restrains macrophage inflammation in adenosine deaminase 2 deficiency

Author(s): Matteo Zoccolillo, Immacolata Brigida, Federica Barzaghi, Serena Scala, Raisa Jofra Hernández, Luca Basso-Ricci, Mariasilvia Colantuoni, Emanuela Pettinato, Lucia Sergi Sergi, Giulia Milardi, Paola Capasso, Angelo Lombardo, Silvia Gregori, Francesca Sanvito, Francesca Schena, Simone Cesaro, Francesca Conti, Andrea Pession, Fabio Benedetti, Marco Gattorno, Pui Y. Lee, Luigi Naldini, Maria Pia Cica
Published in: Blood Advances, Issue 5/16, 2021, Page(s) 3174-3187, ISSN 2473-9529
DOI: 10.1182/bloodadvances.2020003811

Update on Clinical Ex Vivo Hematopoietic Stem Cell Gene Therapy for Inherited Monogenic Diseases

Author(s): Francesca Tucci, Samantha Scaramuzza, Alessandro Aiuti, Alessandra Mortellaro
Published in: Molecular Therapy, Issue 29/2, 2021, Page(s) 489-504, ISSN 1525-0016
DOI: 10.1016/j.ymthe.2020.11.020