Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease

Valentina Poletti (1) , Fabrizia Urbinati , Sabine Charrier , Guillaume Corre (2) , Roger Hollis , Beatriz Campo Fernandez , Samia Martin (3) , Michael Rothe , Axel Schambach , Donald Kohn , Fulvio Mavilio (3)

1 UNIMORE - Università degli Studi di Modena e Reggio Emilia = University of Modena and Reggio Emilia
2 INTEGRARE - Approches génétiques intégrées et nouvelles thérapies pour les maladies rares
3 Généthon

Domains: Cellular Biology; Biochemistry, Molecular Biology; Quantitative Methods [q-bio.QM]; Biotechnology; Genetics; Immunology

Valentina Poletti, Fabrizia Urbinati, Sabine Charrier, Guillaume Corre, Roger Hollis, et al.. Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease. Molecular Therapy - Methods and Clinical Development, 2018, 11, pp.167-179. ⟨10.1016/j.omtm.2018.10.014⟩. ⟨hal-02178306⟩