Published in
Yearbook of Paediatric Endocrinology, 2022
DOI: 10.1530/ey.19.14.14
American Association for the Advancement of Science, Science Advances, 44(7), 2021
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A novel therapeutic strategy for skeletal disorders: Proof of concept of gene therapy for X-linked hypophosphatemia
Journal article published in 2021 by
Volha V. Zhukouskaya 
,
Louisa Jauze ,
Séverine Charles,
Christian Leborgne ,
Stéphane Hilliquin,
Jérémy Sadoine,
Lotfi Slimani,
Brigitte Baroukh,
Laetitia van Wittenberghe,
Wittenberghe Laetitia Van,
Natalie Danièle ,
Fabienne Rajas,
Agnès Linglart ,
Federico Mingozzi ,
Catherine Chaussain
and other authors.
,
Louisa Jauze ,
Séverine Charles,
Christian Leborgne ,
Stéphane Hilliquin,
Jérémy Sadoine,
Lotfi Slimani,
Brigitte Baroukh,
Laetitia van Wittenberghe,
Wittenberghe Laetitia Van,
Natalie Danièle ,
Fabienne Rajas,
Agnès Linglart ,
Federico Mingozzi ,
Catherine Chaussain
and other authors.
This paper is made freely available by the publisher.
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Abstract
A new therapeutic approach for the treatment of a skeletal disorder with AAVs bypasses bone resistance to gene transfer.