Gapmers and methods of using the same for the treatment of muscular dystrophy

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United States of America Patent

PATENT NO 11518995
APP PUB NO 20200291398A1
SERIAL NO

16649122

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Abstract

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The disclosure relates to compositions comprising a nucleotide sequence having two domains: a locked nucleic acid (LNA) domain and a DNA gap domain, wherein nucleotide sequence binds to an endogenous DUX4 mRNA sequence disrupts DUX4 expression.

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Patent Owner(s)

  • CHILDREN'S NATIONAL MEDICAL CENTER; THE GOVERNORS OF THE UNIVERSITY OF ALBERTA

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Inventor(s)

Inventor Name Address # of filed Patents Total Citations
Chen, Yi-wen Washington, US 422 5201
Echigoya, Yusuke Fuji-sawa, JP 4 1
Yokota, Toshifumi Edmonton, CA 5 20
Yokota-Maruyama, Rika Edmonton, CA 1 0

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