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Oligonucleotide Compounds for Targeting Huntingtin Gene

详细技术说明
This new invention stems from the discovery of potent silencing site that was previously unidentified on Huntingtin gene (htt) through screening of more than 200 compounds. Conventional siRNAs will ta
*Abstract

This new invention stems from the discovery of potent silencing site that was previously unidentified on Huntingtin gene (htt) through screening of more than 200 compounds. Conventional siRNAs will target this 3' UTR site for potent inhibition. Efficacy was demonstrated using different siRNA configurations in several cell types and primary neurons that may aid research and future therapeutic applications.

*IP Issue Date
None
*IP Type
Other Patent
国家
Not Available
申请号码
None
国家/地区
美国

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