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Antisense oligonucleotides that target a cryptic splice site in Ush1c as a therapeutic for usher syndrome
Antisense oligonucleotides that target a cryptic splice site in Ush1c as a therapeutic for usher syndrome
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机译:靶向Ush1c中一个隐秘剪接位点的反义寡核苷酸,作为迎来综合征的治疗剂
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摘要
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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