Invention Grant
US09556434B2 Antisense oligonucleotides that target a cryptic splice site in Ush1c as a therapeutic for usher syndrome
有权
反义寡核苷酸靶向Ush1c中的隐性剪接位点，作为引发综合征的治疗剂
- Patent Title: Antisense oligonucleotides that target a cryptic splice site in Ush1c as a therapeutic for usher syndrome
- Patent Title (中): 反义寡核苷酸靶向Ush1c中的隐性剪接位点，作为引发综合征的治疗剂
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Application No.: US14705579Application Date: 2015-05-06
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Publication No.: US09556434B2Publication Date: 2017-01-31
- Inventor: Michelle L Hastings
- Applicant: ROSALIND FRANKLIN UNIVERSITY OF MEDICINE AND SCIENCE
- 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: McDonnell Boehnen Hulbert & Berghoff LLP
- Main IPC: C07H21/04
- IPC: C07H21/04 ; C12N15/113 ; A61K31/712 ; A61K31/7115 ; A61K31/7125
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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