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

CORDIS provides links to public deliverables and publications of HORIZON projects.

Links to deliverables and publications from FP7 projects, as well as links to some specific result types such as dataset and software, are dynamically retrieved from OpenAIRE .

Publications

Lentiviral correction of enzymatic activity restrains macrophage inflammation in adenosine deaminase 2 deficiency (opens in new window)

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
Publisher: The American Society of Hematology
DOI: 10.1182/bloodadvances.2020003811

Update on Clinical Ex Vivo Hematopoietic Stem Cell Gene Therapy for Inherited Monogenic Diseases (opens in new window)

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
Publisher: Nature Publishing Group
DOI: 10.1016/j.ymthe.2020.11.020

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