Correction of osteopetrosis in the neonate oc/oc murine model after lentiviral vector gene therapy and non-genotoxic conditioning
These results will pave the way to the implementation of an effective GT protocol, reducing the transplant-related complication risks in the very young and severely affected ARO patients.
Authors
- Francesca Ficara (ORCID: https://orcid.org/0000-0003-1873-1189)
- Andrea Cappelleri (ORCID: https://orcid.org/0000-0003-3729-528X)
- Eleonora Palagano (ORCID: https://orcid.org/0000-0002-7439-9830)
- Valentina Capo (ORCID: https://orcid.org/0000-0002-1851-1429)
- Luca Basso‐Ricci (ORCID: https://orcid.org/0000-0002-5907-005X)
- Antonella Forlino (ORCID: https://orcid.org/0000-0002-6385-1182)
- Elena Fontana (ORCID: https://orcid.org/0000-0001-8853-3335)
- Stefano Mantero (ORCID: https://orcid.org/0000-0003-0608-2724)
- Eugenio Scanziani (ORCID: https://orcid.org/0000-0003-3996-5048)
- Thorsten Schinke (ORCID: https://orcid.org/0000-0002-8576-0177)
- Marta Filibian (ORCID: https://orcid.org/0000-0003-0939-7879)
- Ludovica Santi (ORCID: https://orcid.org/0000-0001-5425-213X)
- Serena Scala (ORCID: https://orcid.org/0000-0002-8579-3809)
- Cristina Sobacchi (ORCID: https://orcid.org/0000-0002-2684-7184)
- Alessandra Zecchillo (ORCID: https://orcid.org/0000-0003-4142-1162)
- Anna Villa (ORCID: https://orcid.org/0000-0003-4428-9013)
- Martina Di Verniere (ORCID: https://orcid.org/0009-0000-0352-1993)
- Laura Crisafulli (ORCID: https://orcid.org/0000-0002-1393-5652)
- Sara Penna (ORCID: https://orcid.org/0000-0002-5036-3809)
- Valeria Iannello
- Elena Rizzoli
Institutions
- Vita-Salute San Raffaele University (IT)
- Universität Hamburg (DE)
- University of Milan (IT)
- University of Pavia (IT)
- Institute of Biosciences and Bioresources (IT)
- University Medical Center Hamburg-Eppendorf (DE)
- Institute of Genetic and Biomedical Research (IT)
- Fondazione Filarete (IT)
- The San Raffaele Telethon Institute for Gene Therapy (IT)
- Istituti di Ricovero e Cura a Carattere Scientifico (IT)
- IRCCS Humanitas Research Hospital (IT)
- Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele (IT)
- University of Milano-Bicocca (IT)
Publication Details
- Journal
- Frontiers in Endocrinology
- Published
- 2024-09-09
- DOI
- https://doi.org/10.3389/fendo.2024.1450349
- Citations
- 2
- Primary Topic
- Bone Metabolism and Diseases
- Type
- article
- Field-Weighted Citation Impact
- 0.47
Funders
- European Calcified Tissue Society
- Fondazione Telethon
- Ministero della Salute
- Ministero dell'Università e della Ricerca