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公开(公告)号：US11964027B2
公开(公告)日：2024-04-23
申请号：US16606633
申请日：2018-04-20
Applicant: Ospedale San Raffaele S.R.L , Fondazione Telethon
Inventor： Anna Christina Kajaste-Rudnitski , Carolina Petrillo , Bernhard Rudolf Gentner , Luigi Naldini , Pietro Genovese , Giulia Schiroli
IPC: A61K48/00 , C07K7/64 , C12N5/0789 , C12N15/69 , C12N15/86
CPC classification number: A61K48/0008 , C07K7/645 , C12N5/0647 , C12N15/69 , C12N15/86 , C12N2740/15043
Abstract: Use of cyclosporin H (CsH) or a derivative thereof for increasing the efficiency of transduction of an isolated population of cells by a viral vector and/or increasing the efficiency of gene editing of an isolated population of cells when transduced by a viral vector.
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公开(公告)号：US11957747B2
公开(公告)日：2024-04-16
申请号：US17143953
申请日：2021-01-07
Applicant: Ospedale San Raffaele S.r.l. , Fondazione Telethon ETS
Inventor： Alessio Cantore , Angelo Leone Lombardo , Luigi Naldini
CPC classification number: A61K39/12 , C12N7/04 , C12N15/86 , A61K2121/00 , C12N2740/10052 , C12N2740/16041 , C12N2740/16052
Abstract: An enveloped viral particle producer or packaging cell, wherein the cell is genetically engineered to decrease expression of MHC-I on the surface of the cell.
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公开(公告)号：US20230340471A1
公开(公告)日：2023-10-26
申请号：US17963631
申请日：2022-10-11
Applicant: Ospedale San Raffaele S.R.L. , Fondazione Telethon
Inventor： Luigi Naldini , Angelo Leone Lombardo , Angelo Amabile , Alessandro Migliara
CPC classification number: C12N15/113 , C12N9/1007 , C07K14/4703 , C12Y201/01037 , C12Y201/01043 , A61K38/45 , A61K48/005 , A61K35/12 , A61K38/1709 , C12N9/22 , C07K2319/80 , C12N2740/16043 , C12N2310/20 , A61K38/00
Abstract: A product comprising two or more artificial transcription repressors (ATRs), or polynucleotides encoding therefor, selected from groups (a), (b), (c) or (d): (a) an ATR comprising a DNA-binding domain operably linked to a KRAB domain or homologue thereof; (b) an ATR comprising a DNA-binding domain operably linked to a DNMT3A, DNMT3B or DNMT1 domain or homologue thereof; (c) an ATR comprising a DNA-binding domain operably linked to a DNMT3L domain or homologue thereof; and (d) an ATR comprising a DNA-binding domain operably linked to a SETDB1 domain or homologue thereof, wherein at least two of the ATRs are selected from different groups (a), (b), (c) or (d).
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公开(公告)号：US11407996B2
公开(公告)日：2022-08-09
申请号：US16384571
申请日：2019-04-15
Applicant: Ospedale San Raffaele S.r.l. , Fondazione Telethon
Inventor： Alessandra Biffi , Bernhard Rudolf Gentner , Luigi Naldini
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.
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Patent Agency Ranking
公开(公告)号：US20180044670A1
公开(公告)日：2018-02-15
申请号：US15693112
申请日：2017-08-31
Applicant: Ospedale San Raffaele S.R.L.
Inventor： Alessandra Biffi , Bernhard Rudolf Gentner , Luigi Naldini
CPC classification number: C12N15/111 , A61K48/00 , C12N2310/141 , C12N2310/51 , C12N2320/32 , C12N2330/51 , C12N2799/027
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.
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公开(公告)号：US20240342264A1
公开(公告)日：2024-10-17
申请号：US18605229
申请日：2024-03-14
Applicant: Ospedale San Raffaele S.r.I. , Fondazione Telethon ETS
Inventor： Alessio Cantore , Angelo Leone Lombardo , Luigi Naldini
CPC classification number: A61K39/12 , C12N7/04 , C12N15/86 , A61K2121/00 , C12N2740/10052 , C12N2740/16041 , C12N2740/16052
Abstract: An enveloped viral particle producer or packaging cell, wherein the cell is genetically engineered to decrease expression of MHC-I on the surface of the cell.
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公开(公告)号：US12214055B2
公开(公告)日：2025-02-04
申请号：US16758752
申请日：2018-10-24
Applicant: EDITAS MEDICINE, INC. , FONDAZIONE TELETHON ETS , OSPEDALE SAN RAFFAELE
Inventor： Cecillia Cotta-Ramusino , Carrie M. Margulies , Luigi Naldini , Pietro Genovese
IPC: A61K48/00 , A61K39/00 , A61P37/02 , C07K14/475 , C07K14/705 , C12N15/10 , C12N15/113 , C12N15/86 , C12N15/90 , A61K35/17
Abstract: Disclosed herein are genome editing systems and related methods which allow for treatment of Hyper IgM Syndrome, a group of disorders characterized by defective CD40 signaling. The compositions and methods described herein rely on the use of donor templates comprising a CD40L exons to restore proper CD40 signaling and B cell class switch recombination.
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公开(公告)号：US09556438B2
公开(公告)日：2017-01-31
申请号：US14332222
申请日：2014-07-15
Inventor： Luigi Naldini , Brian Brown
IPC: C12N15/11 , C12N15/113 , C12N15/63 , C12N15/86 , A61K48/00
CPC classification number: C12N15/1137 , A61K48/00 , C12N15/635 , C12N15/86 , C12N2310/141 , C12N2740/15043 , C12N2740/15045 , C12N2760/20222 , C12N2810/6081
Abstract: A gene vector comprising a miRNA sequence target.
Abstract translation: 包含miRNA序列靶的基因载体。
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29.发明授权公开(公告)号：US10117899B2
公开(公告)日：2018-11-06
申请号：US14516189
申请日：2014-10-16
Inventor： Pietro Genovese , Philip D. Gregory , Michael C. Holmes , Angelo Leone Lombardo , Luigi Naldini
IPC: C12N5/10 , C12N15/867 , C12N15/90 , C12N5/16 , C12N5/0789 , A61K35/28 , A61K48/00 , C12N9/22 , C12N15/86 , A61K35/12
Abstract: The present disclosure is in the field of genome engineering, particularly targeted modification of the genome of a hematopoietic cell.
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