A novel therapeutic strategy for skeletal disorders: Proof of concept of gene therapy for X-linked hypophosphatemia

Adeno-associated virus (AAV) vectors are a well-established gene transfer approach for rare genetic diseases. Nonetheless, some tissues, such as bone, remain refractory to AAV. X-linked hypophosphatemia (XLH) is a rare skeletal disorder associated with increased levels of fibroblast growth factor 23...

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Detalles Bibliográficos
Autores principales: Zhukouskaya, Volha V., Jauze, Louisa, Charles, Séverine, Leborgne, Christian, Hilliquin, Stéphane, Sadoine, Jérémy, Slimani, Lotfi, Baroukh, Brigitte, van Wittenberghe, Laetitia, Danièle, Natalie, Rajas, Fabienne, Linglart, Agnès, Mingozzi, Federico, Chaussain, Catherine, Bardet, Claire, Ronzitti, Giuseppe
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Association for the Advancement of Science 2021
Materias:
Biomedicine and Life Sciences
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8550245/
https://www.ncbi.nlm.nih.gov/pubmed/34705504
http://dx.doi.org/10.1126/sciadv.abj5018

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