Methods and compositions for treating gain-of-function...

Details Methods and compositions for treating gain-of-function... Methods and compositions for treating gain-of-function...

2011-08-30

2011-08-30

Vivlemore, Tracy (Department: 1635)

Drug, bio-affecting and body treating compositions

Designated organic active ingredient containing

Carbohydrate doai

C536S024500

Reexamination Certificate

active

08008271

ABSTRACT:
The present invention relates to novel methods for treating dominant gain-of-function diseases. The invention provides methods for targeting regions of the copper zinc superoxide dismutase (SOD1), which causes inherited amyotrophic lateral sclerosis (ALS), with RNAi agent. The invention further provides RNAi resistant replacement genes containing mismatches with their respective RNAi agents. The invention also provides for vectors that express RNAi agent and RNAi resistant replacement gene of the present invention.

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