RNA INTERFERENCE FOR THE TREATMENT OF GAIN-OF-FUNCTION DISORDERS
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-func...
Saved in:
Main Authors | , |
---|---|
Format | Patent |
Language | English |
Published |
18.12.2014
|
Subjects | |
Online Access | Get full text |
Cover
Loading…
Summary: | 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. |
---|---|
Bibliography: | Application Number: US201414161266 |