SIRNA IN THE TREATMENT OF ECTRODACTYLY-ECTODERMAL DYSPLASIA-CLEFTING SYNDROME (EEC)

摘要

The invention concerns the pharmacological field, and in particular the treatment of diseases related to the Ectrodactyly-Ectodermal Dysplasia-Clefting Syndrome. A small interfering RNA (siRNA) and use thereof as a medicament are described. A pharmaceutical composition comprising this siRNA, specific for the R279H mutation of the p63 gene, is also described.