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

Project Information

DADA2GT

Grant agreement ID: 841780

DOI 10.3030/841780

Project closed

EC signature date 11 April 2019

Start date 1 June 2019

End date 31 May 2021

Funded under

• EXCELLENT SCIENCE - Marie Skłodowska-Curie Actions

Total cost € 183 473,28

EU contribution € 183 473,28

Coordinated by

OSPEDALE SAN RAFFAELE SRL
Italy

Publications

Peer reviewed articles (2)

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
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

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