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A novel therapeutic strategy for skeletal disorders: Proof of concept of gene therapy for X-linked hypophosphatemia
Volha V. Zhukouskaya, Louisa Jauze, Séverine Charles, Christian Leborgne, Stéphane Hilliquin, Jérémy Sadoine, Lotfi Slimani, Brigitte Baroukh, Laetitia van Wittenberghe, Natalie Danièle, Fabienne Rajas, Agnès Linglart, Federico Mingozzi, Catherine Chaussain, Claire Bardet, Giuseppe Ronzitti
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Source: Crossref
Published: Oct 29, 2021
DOI: 10.1126/sciadv.abj5018
Open original source ↗Source abstract
A new therapeutic approach for the treatment of a skeletal disorder with AAVs bypasses bone resistance to gene transfer.
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