Small interfering RNA (siRNA) for the therapy of type 2 (AD02) autosomal dominant osteopetrosis caused by CLCN7 (AD02 CLCN7-dependent) gene mutation

摘要

The present invention lies in the field of molecules known as "small interfering RNA" with therapeutic applications. siRNAs have the ability to reduce gene expression in an extremely specific way (1). These are small sequences of double-strand RNA, normally used in laboratory to modify cell function, which revolutionized cell biology by allowing previously precluded molecular manipulations.