Invention Publication
- Patent Title: COMPOSITIONS AND METHODS FOR TREATING CYSTIC FIBROSIS
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Application No.: US17911665Application Date: 2021-03-25
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Publication No.: US20230142669A1Publication Date: 2023-05-11
- Inventor: Yifat OREN , Ofra BARCHAD-AVITZUR , Efrat OZERI-GALAI
- Applicant: SpliSense Ltd.
- Applicant Address: IL Jerusalem
- Assignee: SpliSense Ltd.
- Current Assignee: SpliSense Ltd.
- Current Assignee Address: IL Jerusalem
- International Application: PCT/IL2021/050345 2021.03.25
- Date entered country: 2022-09-15
- Main IPC: C12N15/113
- IPC: C12N15/113 ; A61K31/712 ; A61K31/47 ; A61K45/06 ; A61K31/443
Abstract:
The present invention is directed to a method for inducing skipping of exon 24 of the cystic fibrosis transmembrane conductance regulator (CFTR) pre-mRNA. Further, treating cystic fibrosis (CF) using a splicing modulator, such as an antisense oligonucleotide, capable of inducing the skipping of exon 24 of the cystic fibrosis transmembrane conductance regulator (CFTR) pre-mRNA. Also provided are a composition and a kit comprising the splicing modulator.
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