Invention Grant
- Patent Title: Genetically engineered hematopoietic stem cell as a platform for systemic protein expression
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Application No.: US16961483Application Date: 2019-01-11
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Publication No.: US12012613B2Publication Date: 2024-06-18
- Inventor: Mario Amendola , Giulia Pavani
- Applicant: GENETHON , UNIVERSITE D'EVRY-VAL-D'ESSONE , INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM)
- Applicant Address: FR Evry
- Assignee: Genethon,Institut National De La Sante Et De La Recherche Medicale
- Current Assignee: Genethon,Institut National De La Sante Et De La Recherche Medicale
- Current Assignee Address: FR Paris; FR Paris
- Agency: WCF IP
- Priority: EP 305026 2018.01.12
- International Application: PCT/EP2019/050710 2019.01.11
- International Announcement: WO2019/138082A 2019.07.18
- Date entered country: 2020-07-10
- Main IPC: C12N5/0789
- IPC: C12N5/0789 ; A61K35/28 ; C12N9/22 ; C12N15/113

Abstract:
The present invention relates to a genetically modified hematopoietic stem cell comprising, in at least one globin gene comprised in the genome thereof, at least one transgene encoding a therapeutic protein or a therapeutic ribonucleic acid, the said transgene being placed under the control of the endogenous promoter of the said globin gene.
Public/Granted literature
- US20200377857A1 GENETICALLY ENGINEERED HEMATOPOIETIC STEM CELL AS A PLATFORM FOR SYSTEMIC PROTEIN EXPRESSION Public/Granted day:2020-12-03
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