- Patent Title: RNA interference for the treatment of gain-of-function disorders
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Application No.: US16415826Application Date: 2019-05-17
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Publication No.: US11299734B2Publication Date: 2022-04-12
- 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 GPM LLP
- Agent James H. Velema, Esq.; Michael Spellberg, Esq.
- Main IPC: C12N15/113
- IPC: C12N15/113 ; C12N15/11 ; A61K48/00 ; C07H21/02 ; C12N5/07
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
- US20200024599A1 RNA INTERFERENCE FOR THE TREATMENT OF GAIN-OF-FUNCTION DISORDERS Public/Granted day:2020-01-23
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