- Patent Title: Antisense compounds targeting genes associated with cystic fibrosis
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Application No.: US15835698Application Date: 2017-12-08
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Publication No.: US10525076B2Publication Date: 2020-01-07
- 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: A61K31/712
- IPC: A61K31/712 ; C12N15/113

Abstract:
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.
Public/Granted literature
- US20180117073A1 ANTISENSE COMPOUNDS TARGETING GENES ASSOCIATED WITH CYSTIC FIBROSIS Public/Granted day:2018-05-03
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