Invention Grant
- Patent Title: Method of allele specific silencing for the treatment of autosomal dominant Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)
-
Application No.: US16077835Application Date: 2017-02-14
-
Publication No.: US11591602B2Publication Date: 2023-02-28
- Inventor: Silvia G. Priori , Rossana Bongianino , Marco Denegri , Carlo Napolitano
- Applicant: Istituti Clinici Scientifici Maugeri SpA SB
- Applicant Address: IT Pavia
- Assignee: Istituti Clinici Scientifici Maugeri SpA SB
- Current Assignee: Istituti Clinici Scientifici Maugeri SpA SB
- Current Assignee Address: IT Pavia
- Agency: Clark & Elbing LLP
- International Application: PCT/IB2017/050809 WO 20170214
- International Announcement: WO2017/141157 WO 20170824
- Main IPC: C12N15/11
- IPC: C12N15/11 ; C12N15/113 ; A61P9/00 ; A61K9/127 ; C12N15/86
Abstract:
The present invention provides a method for the treatment of autosomal dominant Catecholaminergic Polymorphic Ventricular Tachycardia associated with mutations in the cardiac ryanodine receptor type 2 (RYR2) gene, by the use of an AAV mediated RNA interference approach to induce allele specific silencing of mutant mRNA.
Public/Granted literature
Information query
IPC分类:
