COMPOSITIONS AND METHODS FOR TREATING CYSTIC FIBROSIS

The present invention is directed to a method for treating cystic fibrosis (CF) using a splicing modulator, such as an antisense oligonucleotide, capable of inducing the skipping of exon 23, exon 24, or both, of the cystic fibrosis transmembrane conductance regulator (CFTR) pre-mRNA. Also provided a...

Full description

Saved in:
Bibliographic Details
Main Authors OREN, YIFAT, OZERI-GALAI, EFRAT
Format Patent
LanguageEnglish
French
Published 01.10.2020
Online AccessGet full text

Cover

More Information
Summary:The present invention is directed to a method for treating cystic fibrosis (CF) using a splicing modulator, such as an antisense oligonucleotide, capable of inducing the skipping of exon 23, exon 24, or both, of the cystic fibrosis transmembrane conductance regulator (CFTR) pre-mRNA. Also provided are a composition and a kit comprising the splicing modulator, and a method of producing thereof. La présente invention concerne un procédé de traitement de la fibrose kystique (CF) à l'aide d'un modulateur d'épissage, tel qu'un oligonucléotide antisens, pouvant induire le saut de l'exon 23, de l'exon 24, ou des deux, du pré-ARNm du régulateur de la conductance transmembranaire de la fibrose kystique (CFTR). L'invention concerne également une composition et un kit comprenant le modulateur d'épissage, et un procédé de production de ceux-ci.
Bibliography:Application Number: CA20203129972