Invention Grant
- Patent Title: Gene vector
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Application No.: US16384571Application Date: 2019-04-15
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Publication No.: US11407996B2Publication Date: 2022-08-09
- Inventor: Alessandra Biffi , Bernhard Rudolf Gentner , Luigi Naldini
- Applicant: Ospedale San Raffaele S.r.l. , Fondazione Telethon
- Applicant Address: IT Milan; IT Rome
- Assignee: Ospedale San Raffaele S.r.l.,Fondazione Telethon
- Current Assignee: Ospedale San Raffaele S.r.l.,Fondazione Telethon
- Current Assignee Address: IT Milan; IT Rome
- Agency: Marshall, Gerstein & Borun LLP
- Main IPC: C12N15/11
- IPC: 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.
Information query
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