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1. (WO2018202884) CHEMICALLY MODIFIED MRNA FOR USE IN THE TREATMENT OF A DISEASE ASSOCIATED WITH THE CFTR GENE

Pub. No.:    WO/2018/202884    International Application No.:    PCT/EP2018/061580
Publication Date: Fri Nov 09 00:59:59 CET 2018 International Filing Date: Sat May 05 01:59:59 CEST 2018
IPC: C07K 14/705
A61K 31/7115
Applicants: EBERHARD KARLS UNIVERSITAET TUEBINGEN MEDIZINISCHE FAKULTAET
Inventors: KORMANN, Michael
DEWERTH, Alexander
HAQUE, Ashiqul
ANTONY, Justin S.
Title: CHEMICALLY MODIFIED MRNA FOR USE IN THE TREATMENT OF A DISEASE ASSOCIATED WITH THE CFTR GENE
Abstract:
The present invention relates to a chemically modified m RNA, a nucleic acid molecule encoding the CFTR cm RNA, a vector comprising said nucleic acid molecule, a host cell comprising said vector, a pharmaceutical composition comprising the CFTR cm RNA, nucleic acid molecule or vector, and to a method for the treatment of a disease associated with the CFTR gene.