专利检索 ap:("CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE" OR "INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM)") AND inv:"Charbel Massaad" 第 1 页

1.

发明授权
Antisense RNA targeting PMP22 for the treatment of Charcot-Marie-Tooth 1A disease 有权

公开(公告)号：US11939577B2

公开(公告)日：2024-03-26

申请号：US17279206

申请日：2019-09-24

申请人： CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE , INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM)

发明人： Liliane Massade , Charbel Massaad , Susan Boutary , Giorgia Maria Laura Urbinati , Patrick Couvreur , Didier Desmaële

IPC分类号： C12N15/113 , A61K9/51 , A61K45/06 , A61K47/69

CPC分类号： C12N15/113 , A61K9/5123 , A61K45/06 , A61K47/6929 , C12N2310/11 , C12N2310/14 , C12N2310/351 , C12N2320/32

摘要： The present invention relates to antisense RNAs targeting PMP22 and able to inhibit from 40% to 60% the expression of PMP22 in the cells and a pharmaceutical composition comprising thereof. The antisense RNAs are preferably siRNA and are preferably provided in the form of nanoparticles. The present invention also relates to the use of these antisense RNAs targeting PMP22 for the treatment of Charcot-Marie-Tooth 1A disease.