Invention Grant
US08648053B2 Antisense oligonucleotides that target a cryptic splice site in Ush1c as a therapeutic for Usher syndrome
有权
靶向Ush1c中的隐性剪接位点的反义寡核苷酸作为Usher综合征的治疗剂
- Patent Title: Antisense oligonucleotides that target a cryptic splice site in Ush1c as a therapeutic for Usher syndrome
- Patent Title (中): 靶向Ush1c中的隐性剪接位点的反义寡核苷酸作为Usher综合征的治疗剂
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Application No.: US13461565Application Date: 2012-05-01
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Publication No.: US08648053B2Publication Date: 2014-02-11
- Inventor: Michelle L. Hastings
- Applicant: Michelle L. Hastings
- Applicant Address: US IL North Chicago
- Assignee: Rosalind Franklin University of Medicine and Science
- Current Assignee: Rosalind Franklin University of Medicine and Science
- Current Assignee Address: US IL North Chicago
- Agency: Ungaretti & Harris LLP
- Agent Joseph A. Fuche
- Main IPC: A61K48/00
- IPC: A61K48/00
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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