Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency

Valentina Poletti (1) , Sabine Charrier , Guillaume Corre (2) , Bernard Gjata (3) , Alban Vignaud (4) , Fang Zhang (5) , Michael Rothe , Axel Schambach , H. Bobby Gaspar , Adrian J. Thrasher (6) , 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
4 Coeur, Muscle et Vaisseaux
5 UCL - University College of London [London]
6 Molecular Immunology Unit

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

Valentina Poletti, Sabine Charrier, Guillaume Corre, Bernard Gjata, Alban Vignaud, et al.. Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency. Molecular Therapy - Methods and Clinical Development, 2018, 9, pp.257-269. ⟨10.1016/j.omtm.2018.03.002⟩. ⟨hal-02178311⟩