Antisense oligonucleotides that target a cryptic splice site in Ush1c as a therapeutic for usher syndrome

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United States of America Patent

PATENT NO 9556434
APP PUB NO 20150315590A1
SERIAL NO

14705579

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Abstract

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The present invention provides a method for treating Usher's syndrome in a human subject including administering to the human subject an oligonucleotide having 8 to 30 linked nucleosides having a nucleobase sequence comprising a complementary region comprising at least 8 contiguous nucleobases complementary to a target region of equal length within exon 3 of an Usher RNA transcript.

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Patent Owner(s)

Patent OwnerAddress
ROSALIND FRANKLIN UNIVERSITY OF MEDICINE AND SCIENCE3333 GREEN BAY ROAD NORTH CHICAGO IL 60064

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Inventor(s)

Inventor Name Address # of filed Patents Total Citations
Hastings, Michelle L Lake Bluff, US 19 40

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