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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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公开(公告)号:US20220241241A1
公开(公告)日:2022-08-04
申请号:US17611777
申请日:2020-05-25
Applicant: ISTITUTO NAZIONALE DI GENETICA MOLECOLARE - INGM , OSPEDALE SAN RAFFAELE S.R.L. , IRBM S.P.A.
Inventor: Raffaele DE FRANCESCO , Lorena DONNICI , Luca GUIDOTTI , Matteo IANNACONE , Romano DI FABIO , Vincenzo SUMMA , Adolfo PRANDI , Pietro RANDAZZO , Leda IVANOVA BENCHEVA , Marilenia DE MATTEO , Luca FERRANTE , Davide GORNATI , Alessandro GRILLO
IPC: A61K31/407 , C07D515/14 , C07D513/14 , A61P31/20 , A61K39/395 , A61K38/21 , A61K31/522 , A61K31/513 , A61K31/7072 , A61K31/675 , A61K31/437 , A61K31/7084 , A61K31/506 , A61K31/445 , A61K39/29
Abstract: The present invention relates to compounds that are inhibitors of hepatitis B virus (HBV). Compounds of this invention are useful alone or in combination with other agents for treating, ameliorating, preventing or curing HBV infection and related conditions. The present invention also relates to pharmaceutical compositions containing the compounds.
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公开(公告)号:US20220119477A1
公开(公告)日:2022-04-21
申请号:US17288406
申请日:2019-10-31
Applicant: Ospedale San Raffaele S.R.L.
Inventor: Maria Chiara Bonini , Eliana Eliana , Zulma Irene Magnani , Fabio Ciceri , Erica Carnevale
IPC: C07K14/725 , C12N15/90 , A61K35/17 , A61K38/00
Abstract: A T-cell receptor (TCR), which binds to a Wilms tumour 1 protein (WT1) peptide when presented by a major histocompatibility complex (MHC).
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公开(公告)号:US20220033849A1
公开(公告)日:2022-02-03
申请号:US17356980
申请日:2021-06-24
Inventor: Mukesh Mayani , Tongyao Liu , Ayman Ismail
IPC: C12N15/86
Abstract: Viral vector production processes and methods of purifying a viral vector from a host cell are provided herein.
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公开(公告)号:US20210252275A1
公开(公告)日:2021-08-19
申请号:US17275886
申请日:2019-09-13
Applicant: OSPEDALE SAN RAFFAELE S.R.L.
Inventor: Federico ESPOSTI , Filippo CONSOLO , Federico Severine PAPPALARDO
IPC: A61M60/515 , A61M60/148
Abstract: The present invention relates to a method for early detection of artificial pump dysfunction based on a time-frequency analysis of the pump motor power consumption (PRC). The method allows to prevent low output syndrome, cardiogenic shock, pump thrombus and/or cardiac arrest or death as well as to monitor the efficacy of a thrombolytic therapy and/or to optimize intensity and duration of a thrombolytic therapy.
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公开(公告)号:US20200038439A1
公开(公告)日:2020-02-06
申请号:US16341704
申请日:2017-10-16
Applicant: CHILDREN'S MEDICAL CENTER CORPORATION , OSPEDALE SAN RAFFAELE S.R.L. , POLITECNICO DI MILANO , FONDAZIONE TELETHON
Inventor: ALESSANDRA BIFFI , MARCO PEVIANI , DAVIDE MOSCATELLI , ALESSIA CAPATANDO , RITA MILAZZO , UMBERTO CAPASSO PALMIERO
Abstract: The present invention provides compositions and methods for the treatment or prevention of a neurological disease or disorder of the central nervous system (e.g., a storage disorder, lysosomal storage disorder, neurodegenerative disease, etc.) by reconstitution of brain myeloid cell and microglia upon transplantation of hematopoietic cells enriched in microglia reconstitution potential. The invention also provides compositions and methods for ablating and reconstituting microglia.
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公开(公告)号:US09944899B2
公开(公告)日:2018-04-17
申请号:US14926761
申请日:2015-10-29
Applicant: OSPEDALE SAN RAFFAELE S.R.L. , Fondazione Telethon
Inventor: Silvia Adriana Gregori , Maria Grazia Roncarolo , Rosa Bacchetta
IPC: C12N5/0783 , C12N5/0784 , A61K35/17 , A01N63/02 , C12N5/071 , A61K35/12
CPC classification number: C12N5/0637 , A61K35/17 , A61K2035/122 , A61K2035/124 , C12N5/0636 , C12N5/064 , C12N2501/22 , C12N2501/23 , C12N2501/231 , C12N2502/1121 , C12N2506/115
Abstract: The present invention relates to a tolerogenic dendritic cell population (Tr-DC) capable of generating a population of T cells having regulatory activity, method of production and uses thereof. Furthermore, soluble HLA-G promotes the differentiation of a population of T cells with regulatory activity.
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公开(公告)号:US20170056558A1
公开(公告)日:2017-03-02
申请号:US15306682
申请日:2015-04-24
Applicant: Ospedale San Raffaele S.r.l. , Fondazione Telethon
Inventor: Anna Kajaste-Rudnitski , Eugenio Montini
CPC classification number: A61L27/3834 , A61K35/28 , A61L27/3608 , A61L27/3616 , A61L27/3687 , C07K7/645 , C12N5/0647 , C12N15/86 , C12N2510/00 , C12N2740/15043 , C12N2740/16043
Abstract: Use of cyclosporin A (CsA) or a derivative thereof for increasing the efficiency of transduction of an isolated population of human haematopoietic stem and/or progenitor cells by a vector derived from HIV-1, HIV-2, FIV, BW, EIAV, CAEV or visna lentivirus.
Abstract translation: 使用环孢菌素A(CsA)或其衍生物通过衍生自HIV-1,HIV-2,FIV,BW,EIAV,CAEV的载体增加分离的人类造血干细胞和/或祖细胞群的转导效率 或visna慢病毒。
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公开(公告)号:US20150203551A1
公开(公告)日:2015-07-23
申请号:US14416128
申请日:2013-07-26
Inventor: Marco Emilio Bianchi , Maura Casalgrandi , Emilie Joelle Venereau , Silvia Brunelli
CPC classification number: C07K14/47 , A61K38/005 , A61K38/1709
Abstract: The present invention relates to HMGB1 variants that maintain HMGB1 wild type chemoattractant function while displaying abolished cytokine and/or chemokine stimulating properties. Such molecules are useful in therapy.
Abstract translation: 本发明涉及在显示消除的细胞因子和/或趋化因子刺激性质的同时保持HMGB1野生型化学引诱物功能的HMGB1变体。 这样的分子可用于治疗。
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公开(公告)号:US12173307B2
公开(公告)日:2024-12-24
申请号:US17356980
申请日:2021-06-24
Inventor: Mukesh Mayani , Tongyao Liu , Ayman Ismail
IPC: C12N15/86
Abstract: Viral vector production processes and methods of purifying a viral vector from a host cell are provided herein.
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