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
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Issued Date -
Aug 28, 2014
app pub date -
Feb 10, 2014
filing date -
Oct 20, 2010
priority date (Note) -
Abandoned
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Abstract
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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