Methods and compositions for treating pachyonychia congenita

摘要

A method for treating pachyonychia congenita (PC) by identifying a genetic mutation contributing to PC, preparing a siRNA sequence that inhibits expression of the identified mutation, and administering the siRNA to a cell of a subject afflicted with PC. The siRNA treatment can include silencing of both the mutated and associated wildtype genes for a specific keratin. Specific mutations and inhibitory sequences are identified.