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公开(公告)号：US20250049888A1
公开(公告)日：2025-02-13
申请号：US18720686
申请日：2022-12-21
Inventor： Giovanni Piccoli , Francesca Pischedda
Abstract: The invention relates to novel Negr1 protein fragments having ALK-inhibiting or ALK-reducing activity and to the nucleic acids encoding therefor, as well as to the use of the Negr1 protein, its fragments and the nucleic acids encoding therefor in the therapeutic treatment of ALK-related diseases such as cancer and tauopathies.
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公开(公告)号：US12076332B2
公开(公告)日：2024-09-03
申请号：US17050406
申请日：2019-04-25
Inventor： Gal Bitan , Alessandro Fraldi , Irene Sambri , Antonio Monaco
IPC: A61K31/663 , A61K31/10 , A61K31/19 , A61K31/6615 , A61P25/00
CPC classification number: A61K31/663 , A61K31/10 , A61K31/19 , A61K31/6615 , A61P25/00
Abstract: In various embodiments methods are provided for the treatment or prophylaxis of liposomal storage diseases. In certain embodiments the methods involve administering to a subject in need thereof one or more molecular tweezers that inhibit protein aggregation.
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3.发明公开公开(公告)号：US20240269169A1
公开(公告)日：2024-08-15
申请号：US18401135
申请日：2023-12-29
Applicant: The Children's Medical Center Corporation , Ospedale San Raffaele S.r.l. , Politecnico di Milano , Fondazione Telethon ETS , Dana-Farber Cancer Institute, Inc.
Inventor： Alessandra BIFFI , Marco PEVIANI , Davide MOSCATELLI , Alessia CAPOTONDO , Rita MILAZZO , Umberto CAPASSO PALMIERO
CPC classification number: A61K35/12 , A61P25/28 , C12N15/86 , A01K67/0271 , A01K2227/10 , A61K9/0019 , A61K2035/124 , A61K45/06 , C12N2740/15043
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：US20240247235A1
公开(公告)日：2024-07-25
申请号：US16972379
申请日：2019-06-06
Applicant: UNIVERSITA' DEGLI STUDI DI PADOVA , FONDAZIONE TELETHON
Inventor： Graziano MARTELLO , Nicola ELVASSORE , Marco PELLEGRINI , Stefano GIULITTI
IPC: C12N5/074
CPC classification number: C12N5/0696 , C12N2501/602 , C12N2501/603 , C12N2501/604 , C12N2501/605 , C12N2501/606 , C12N2501/727 , C12N2506/1307
Abstract: A method obtains naive induced iPSCs (niPSCs) from mammalian somatic cells. The method includes a transgene cocktail delivery to somatic cells in a microfluidic setting, including a first step of Mesenchymal to Epithelial Transition (MET) and a second step of colonies generation.
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公开(公告)号：US20240218370A1
公开(公告)日：2024-07-04
申请号：US18191611
申请日：2023-03-28
Applicant: OSPEDALE SAN RAFFAELE S.R.L. , Fondazione Telethon
Inventor： Luigi Naldini , Brian Brown
IPC: C12N15/113 , C12N15/63 , C12N15/86
CPC classification number: C12N15/1137 , C12N15/635 , C12N15/86 , A61K48/00
Abstract: A gene vector comprising a miRNA sequence target.
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Patent Agency Ranking
公开(公告)号：US20220380807A1
公开(公告)日：2022-12-01
申请号：US17861676
申请日：2022-07-11
Applicant: INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) , UNIVERSITÉ D'EVRY-VAL-D'ESSONNE , GENETHON , FONDAZIONE ISTITUTO ITALIANO DI TECNOLOGIA , FONDAZIONE TELETHON
Inventor： Federico Mingozzi , Giuseppe Ronzitti , Andrea Contestabile , Laura Cancedda
IPC: C12N15/86 , A61K45/06 , C12N15/113
Abstract: The invention relates to an RNA interference (RNAi) strategy based on the use of artificial microRNA (amiR) to reduce NKCC1 expression. In particular, the invention relates to a vector that achieves neuron-specific expression of specific amiR against NKCC1 by using a human Synapsin promoter to drive transgene expression.
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公开(公告)号：US20220273712A1
公开(公告)日：2022-09-01
申请号：US17615766
申请日：2020-06-05
Applicant: THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY , OSPEDALE SAN RAFFAELE S.R.L. , FONDAZIONE TELETHON
Inventor： Rosa Bacchetta , Maria Grazia Roncarolo , Yohei Sato , Luigi Naldini , Laura Passerini
IPC: A61K35/17 , C12N5/0783
Abstract: Engineered Treg-like cells, CD4LVFOXP3 T cells, and their use in cellular therapy to promote immune tolerance are disclosed. In particular, CD4LVFOXP3 T cells are produced by transduction of CD4+ T cells with a lentiviral vector expressing FOXP3 under the control of a constitutive promoter. Transduced cells express FOXP3 at high and persistent levels and acquire immune suppressive characteristics resembling naturally occurring Treg cells.
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公开(公告)号：US20220002749A1
公开(公告)日：2022-01-06
申请号：US17202098
申请日：2021-03-15
Applicant: Fondazione Telethon (IT/IT)
Inventor： Pasqualina Colella , Alberto Auricchio , Ivana Trapani
IPC: C12N15/86 , C07K14/705
Abstract: The present invention relates to constructs, vectors, relative host cells and pharmaceutical compositions which allow an effective gene therapy, in particular of genes larger than 5 Kb.
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公开(公告)号：US20210371878A1
公开(公告)日：2021-12-02
申请号：US17285356
申请日：2019-10-15
Applicant: FONDAZIONE TELETHON
Inventor： Alberto AURICCHIO , Ivana TRAPANI , Patrizia TORNABENE
Abstract: The present invention relates to constructs, vectors, relative host cells and pharmaceutical compositions which allow an effective gene therapy, in particular of genes larger than 5 Kb.
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公开(公告)号：US20210317411A1
公开(公告)日：2021-10-14
申请号：US17250050
申请日：2019-05-16
Applicant: Ospedale San Raffaele S.r.l. , Fondazione Telethon
Inventor： Claire LATROCHE , Luigi NALDINI , Maura MANZI
IPC: C12N5/0789 , A61K35/12
Abstract: Use of CD47 and/or C-X-C chemokine receptor type 4 (CXCR4) for increasing engraftment by haematopoietic stem C and/or progenitor cells (HSPCs).
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