Supported projects

AA202001 – Gene therapy for X-linked hypophosphatemia

Scientific leader
Dr. Claire Bardet

X-linked hypophosphatemia (XLH) is the most common vitamin-resistant hypophosphatemic rickets of genetic origin (1/20000) where elevated levels of FGF23 are the critical event leading to chronic hypophosphatemia associated with skeletal manifestations. A recent strategy aims to neutralize the action of FGF23 by using FGF23 monoclonal antibodies with the advantage of normalizing phosphatemia and regulating other pathological parameters. We propose to develop an innovative gene therapy approach to treat XLH using a single injection based on the use of a liver-targeted vector to produce a factor neutralizing FGF23, the main factor in origin of damage to the skeleton. Our project opens up new perspectives in skeletal disease treatments using liver gene therapy.



Laboratoire UR 2496 Pathologies, Imagerie et Biothérapie Orofaciales

Université de Paris

1, rue Maurice Arnoux

92 120 Paris