Invention Grant
- Patent Title: Synthetic polynucleotides encoding a human FKRP protein
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Application No.: US16628650Application Date: 2018-07-06
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Publication No.: US11596698B2Publication Date: 2023-03-07
- Inventor: Isabelle Richard , Evelyne Gicquel-Zouida , William Lostal
- Applicant: GENETHON , UNIVERSITE D'EVRY-VAL-D'ESSONNE , INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE)
- Applicant Address: FR Evry; FR Evry; FR Paris
- Assignee: GENETHON,UNIVERSITE D'EVRY-VAL-D'ESSONNE,INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE)
- Current Assignee: GENETHON,UNIVERSITE D'EVRY-VAL-D'ESSONNE,INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE)
- Current Assignee Address: FR Evry; FR Evry; FR Paris
- Agency: Knobbe, Martens, Olson & Bear LLP
- Priority: EP17305894 20170707
- International Application: PCT/EP2018/068420 WO 20180706
- International Announcement: WO2019/008157 WO 20190110
- Main IPC: A61K48/00
- IPC: A61K48/00 ; A61P21/00 ; C07K14/47 ; C12N15/86 ; A61K38/00
Abstract:
The present invention concerns synthetic polynucleotides encoding a human fukutin-related protein (FKRP) wherein the synthetic polynucleotides contain at least a mutation avoiding supplementary transcript(s) generated from frameshift start codon(s). The synthetic polynucleotides are useful, especially for treating a pathology linked to a FKRP deficiency or induced by a defect in α-dystroglycan (α-DG) glycosylation, such as LGMD2I.
Public/Granted literature
- US20210338837A1 NOVEL POLYNUCLEOTIDES ENCODING A HUMAN FKRP PROTEIN Public/Granted day:2021-11-04
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