- 专利标题: Antisense compounds targeting genes associated with cystic fibrosis
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申请号: US15835995申请日: 2017-12-08
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公开(公告)号: US10544417B2公开(公告)日: 2020-01-28
- 发明人: Michelle L. Hastings
- 申请人: Rosalind Franklin University of Medicine and Science
- 申请人地址: US IL North Chicago
- 专利权人: Rosalind Franklin University of Medicine and Science
- 当前专利权人: Rosalind Franklin University of Medicine and Science
- 当前专利权人地址: US IL North Chicago
- 代理机构: McDonnell Boehnen Hulbert & Berghoff LLP
- 主分类号: C12N15/113
- IPC分类号: C12N15/113
摘要:
The present disclosure relates generally to compounds comprising oligonucleotides complementary to a cystic fibrosis transmembrane conductance regulator (CFTR) RNA transcript. Certain such compounds are useful for hybridizing to a CFTR RNA transcript, including but not limited to a CFTR RNA transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the CFTR transcript. In certain embodiments, such compounds are used to treat one or more symptoms associated with Cystic Fibrosis.
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