hrp0092rfc15.1 | Late Breaking Abstracts | ESPE2019
Le Stunff Catherine
, Gunes Yasemin
, Mille Clémence
, Bougnères Pierre
The use of recombinant adeno-associated viruses (rAAV) as safe vectors have allowed hundreds of gene therapy attempts to treat monogenic diseases not including bone genetic diseases (Gao G, Nat Rev Drug Dis 2019). To our knowledge, there has been few attempts to apply gene therapy to monogenic bone diseases, largely because most skeletal malformations are being developed during fetal life. Patients affected with acrodysostosis are known to aggravate their skeletal malformation...