Martina Di Verniere

Partial correction of immunodeficiency by lentiviral vector gene therapy in mouse models carrying Rag1 hypomorphic mutations

Valentina Capo, M. C. Castiello, P. Uva, 2023, Frontiers in immunology.

Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency

C. Brombin, G. Crooks, Samuele Ferrari, 2024, Science Translational Medicine.