Nucleoside agents for the reduction of the deleterious activity of extended nucleotide repeat containing genes

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United States of America Patent

PATENT NO 10675293
APP PUB NO 20180064744A1
SERIAL NO

15554679

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Abstract

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Aspects of the invention include methods of reducing the deleterious activity of a mutant extended nucleotide repeat (NR) containing target gene in a cell by contacting the cell with an effective amount of a nucleoside agent, as well as compositions used in such methods. The deleterious activity (e.g., toxicity and/or dis-functionality of products encoded thereby) of a mutant extended NR containing target gene may be reduced in a variety of different ways, e.g., by reducing (and in some instances differentially, including selectively, reducing) the production or activity of toxic expression products (e.g., RNA or protein) encoded by the target gene. Kits and compositions for practicing the subject methods are also provided.

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Patent Owner(s)

Patent OwnerAddress
THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITYOFFICE OF THE GENERAL COUNSEL BUILDING 170 THIRD FLOOR MAIN QUAD PO BOX 20386 STANFORD CA 94305-2038

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Inventor(s)

Inventor Name Address # of filed Patents Total Citations
Cheng, Tzu-Hao Taipei, TW 11 42
Cohen, Stanley N Stanford, US 51 1002
Deng, Ning Palo Alto, US 34 189
Feng, Yanan San Jose, US 4 3
Hsieh, Wen-Chieh Taipei, TW 8 23
Wu, Yun-Yun Taipei, TW 3 10

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