Penna S, Zecchillo A, Di Verniere M, Fontana E, Iannello V, Palagano E, Mantero S, Cappelleri A, Rizzoli E, Santi L, Crisafulli L, Filibian M, Forlino A, Basso-Ricci L, Scala S, Scanziani E, Schinke T, Ficara F, Sobacchi C, Villa A, Capo V (2024) Correction of osteopetrosis in the neonate oc/oc murine model after lentiviral vector gene therapy and non-genotoxic conditioning. Front Endocrinol (Lausanne) 15:1450349. doi: 10.3389/fendo.2024.1450349 PMID: 39314524
Objective: To investigate whether gene therapy (GT) could minimize the immune-mediated complications of allogeneic hematopoietic stem cell transplantation (HSCT) and offer a prompt treatment.
Summary: Authors showed that lentiviral vector GT can revert the osteopetrotic bone phenotype, allowing long-term survival and reducing extramedullary haematopoiesis and that plerixafor-induced mobilization can further increase the high number of HSPCs circulating in peripheral blood, facilitating the collection of adequate numbers of cells for therapeutic purposes. Pre-transplant non-genotoxic conditioning allowed the stable engraftment of HSPCs, albeit at lower level than conventional total body irradiation, and led to long-term survival and correction of bone phenotype, in the absence of acute toxicity.
Usage: Recipient oc/oc mice were conditioned by CD45-SAP injections or total body irradiation (IRR)
Related Products: Anti-CD45.2-SAP (Cat. #IT-91)