- Patent Title: RNA interference for the treatment of gain-of-function disorders
-
Application No.: US15226476Application Date: 2016-08-02
-
Publication No.: US10344277B2Publication Date: 2019-07-09
- Inventor: Neil Aronin , Phillip D. Zamore
- Applicant: University of Massachusetts
- Applicant Address: US MA Boston
- Assignee: UNIVERSITY OF MASSACHUSETTS
- Current Assignee: UNIVERSITY OF MASSACHUSETTS
- Current Assignee Address: US MA Boston
- Agency: Lathrop Gage LLP
- Agent James H. Velema, Esq.
- Main IPC: C12N15/63
- IPC: C12N15/63 ; C12N15/113 ; A61K48/00 ; C12N15/11
Abstract:
The present invention relates to the discovery of an effective treatment for a variety of gain-of-function diseases, in particular, Huntington's disease (HD). The present invention utilizes RNA Interference technology (RNAi) against polymorphic regions in the genes encoding various gain-of-function mutant proteins resulting in an effective treatment for the gain-of-function disease.
Public/Granted literature
- US20170051283A1 RNA INTERFERENCE FOR THE TREATMENT OF GAIN-OF-FUNCTION DISORDERS Public/Granted day:2017-02-23
Information query
IPC分类:
