ARN DE INTERFERÊNCIA PEQUENO (ARNSI) PARA A TERAPÊUTICA DA OSTEOPETROSE AUTOSSÓMICA DOMINANTE DE TIPO 2 (ADO2) CAUSADA POR MUTAÇÃO GÉNICA DE CLCN7 (ADO2 DEPENDENTE DE CLCN7)
ANNA MARIA TETI,NADIA RUCCI,MATTIA CAPULLI,ANTONIO MAURIZI
申请号：
PT15732387
公开号：
PT3145553T
申请日:
2015.05.21
申请国别(地区):
PT
年份:
2019
代理人:
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.
