Invention Application
- Patent Title: GENE VECTOR
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Application No.: US17855135Application Date: 2022-06-30
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Publication No.: US20220333103A1Publication Date: 2022-10-20
- 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
- Main IPC: C12N15/11
- IPC: C12N15/11
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
IPC分类:
