Expanding and extending gene therapy of monogenic diseases of the haematopoietic system

Project Information

GENE FOR CURE

Grant agreement ID: 693762

DOI 10.3030/693762

EC signature date 13 December 2016

Start date 1 October 2016

End date 30 September 2024

Funded under

• EXCELLENT SCIENCE - European Research Council (ERC)

Total cost € 2 445 267,52

EU contribution € 2 445 267,52

Coordinated by

INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
France

Publications

Peer reviewed articles (3)

Gene Therapy for β-Hemoglobinopathies

Author(s): Marina Cavazzana, Chiara Antoniani, Annarita Miccio
Published in: Molecular Therapy, Issue 25/5, 2017, Page(s) 1142-1154, ISSN 1525-0016
Publisher: Nature Publishing Group
DOI: 10.1016/j.ymthe.2017.03.024

Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus

Author(s): Chiara Antoniani, Vasco Meneghini, Annalisa Lattanzi, Tristan Felix, Oriana Romano, Elisa Magrin, Leslie Weber, Giulia Pavani, Sara El Hoss, Ryo Kurita, Yukio Nakamura, Thomas J. Cradick, Ante S. Lundberg, Matthew Porteus, Mario Amendola, Wassim El Nemer, Marina Cavazzana, Fulvio Mavilio, Annarita Miccio
Published in: Blood, Issue 131/17, 2018, Page(s) 1960-1973, ISSN 0006-4971
Publisher: American Society of Hematology
DOI: 10.1182/blood-2017-10-811505

An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype

Author(s): Leslie Weber, Valentina Poletti, Elisa Magrin, Chiara Antoniani, Samia Martin, Charles Bayard, Hanem Sadek, Tristan Felix, Vasco Meneghini, Michael N. Antoniou, Wassim El-Nemer, Fulvio Mavilio, Marina Cavazzana, Isabelle Andre-Schmutz, Annarita Miccio
Published in: Molecular Therapy - Methods & Clinical Development, Issue 10, 2018, Page(s) 268-280, ISSN 2329-0501
Publisher: Science Direct
DOI: 10.1016/j.omtm.2018.07.012