Correction of osteopetrosis in the neonate oc/oc murine model after lentiviral vector gene therapy and non-genotoxic conditioning

IntroductionAutosomal recessive osteopetrosis (ARO) is a rare genetic disease, characterized by increased bone density due to defective osteoclast function. Most of the cases are due to TCIRG1 gene mutation, leading to severe bone phenotype and death in the first years of life. The standard therapy...

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Principais autores:	Sara Penna, Alessandra Zecchillo, Martina Di Verniere, Elena Fontana, Valeria Iannello, Eleonora Palagano, Stefano Mantero, Andrea Cappelleri, Elena Rizzoli, Ludovica Santi, Laura Crisafulli, Marta Filibian, Antonella Forlino, Luca Basso-Ricci, Serena Scala, Eugenio Scanziani, Thorsten Schinke, Francesca Ficara, Cristina Sobacchi, Anna Villa, Valentina Capo
Formato:	Artigo
Idioma:	Inglês
Publicado em:	Frontiers Media S.A. 2024-09-01
Colecção:	Frontiers in Endocrinology
Assuntos:	gene therapy osteopetrosis lentiviral vector osteoclast hematopoietic stem cells HSC mobilization
Acesso em linha:	https://www.frontiersin.org/articles/10.3389/fendo.2024.1450349/full
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