Invention Grant
- Patent Title: Gene vector
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Application No.: US15693112Application Date: 2017-08-31
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Publication No.: US09951328B2Publication Date: 2018-04-24
- Inventor: Alessandra Biffi , Bernhard Rudolf Gentner , Luigi Naldini
- Applicant: Fondazione Telethon , Ospedale San Raffaele S.r.l.
- Applicant Address: IT Rome IT Milan
- Assignee: Fondazione Telethon,Ospedale San Raffaele S.r.l.
- Current Assignee: Fondazione Telethon,Ospedale San Raffaele S.r.l.
- Current Assignee Address: IT Rome IT Milan
- Agency: Marshall, Gerstein & Borun LLP
- Main IPC: C12N15/63
- IPC: C12N15/63 ; C12N15/113 ; C12N15/86 ; C12N15/11 ; A61K48/00

Abstract:
A gene vector for use in gene therapy comprising at least one miRNA sequence target operably linked to a nucleotide sequence having a corresponding miRNA in a hematopoietic progenitor cell (HSPC) or hematopoietic stem cell (HSC) which prevents or reduces expression of the nucleotide sequence in a HSPC or HSC but not in a differentiated cell.
Public/Granted literature
- US20180044670A1 GENE VECTOR Public/Granted day:2018-02-15
Information query
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