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公开(公告)号：US20240009247A1
公开(公告)日：2024-01-11
申请号：US18342642
申请日：2023-06-27
Applicant: The Regents of the University of California
Inventor： Stephanie Cherqui , Eric Adler , Sylvia Evans
IPC: A61K35/28 , A61P3/00 , A61K35/14 , C07K14/705 , C12N15/86
Abstract: Provided herein are methods for treating a lysosomal transmembrane protein disease or disorder through ex vivo introduction of a nucleic acid molecule into hematopoietic stem and progenitor cells (HSPCs) followed by transplantation of the HSPCs into a subject in need of treatment. Also provided are vectors containing the nucleic acid molecule.
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公开(公告)号：US20210161966A1
公开(公告)日：2021-06-03
申请号：US16493573
申请日：2018-03-15
Applicant: The Regents of the University of California
Inventor： Stephanie Cherqui , Eric Adler , Sylvia Evans
IPC: A61K35/28 , A61P3/00 , A61K35/14 , C12N15/86 , C07K14/705
Abstract: Provided herein are methods for treating a lysosomal transmembrane protein disease or disorder through ex vivo introduction of a nucleic acid molecule into hematopoietic stem and progenitor cells (HSPCs) followed by transplantation of the HSPCs into a subject in need of treatment. Also provided are vectors containing the nucleic acid molecule.
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公开(公告)号：US20240301017A1
公开(公告)日：2024-09-12
申请号：US18658138
申请日：2024-05-08
Applicant: The Regents of the University of California
Inventor： Stephanie Cherqui
IPC: C07K14/47 , A01K67/0276 , A01K67/0278 , A61K35/28 , A61P25/28 , C12N9/22 , C12N15/11 , C12N15/86
CPC classification number: C07K14/47 , A01K67/0276 , A01K67/0278 , A61K35/28 , A61P25/28 , C12N9/22 , C12N15/11 , C12N15/86 , A01K2207/15 , A01K2217/05 , A01K2217/075 , A01K2217/15 , A01K2227/105 , A01K2267/0318 , C07K2319/60 , C12N2310/20 , C12N2740/16043 , C12N2800/80
Abstract: Provided herein are methods for treating a disease or disorder associated with mitochondrial dysfunction through ex vivo introduction of a nucleic acid molecule into hematopoietic stem and progenitor cells (HSPCs) followed by transplantation of the HSPCs into a subject in need of treatment. The nucleic acid molecule may include a functional human frataxin (hFXN) or may include a gene editing system that when transfected into the cells removes a trinucleotide extension mutation of endogenous hFXN.
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公开(公告)号：US20220177918A1
公开(公告)日：2022-06-09
申请号：US17602987
申请日：2020-04-07
Applicant: The Regents of the University of California
Inventor： Stephanie Cherqui
IPC: C12N15/86 , C07K14/47 , A61P27/02 , A61K35/761
Abstract: Provided herein are methods for treating an inherited eye disease or disorder through ex vivo introduction of a nucleic acid molecule into hematopoietic stem and progenitor cells (HSPCs) followed by transplantation of the HSPCs into a subject's eyes in need of treatment. Also provided are vectors containing the nucleic acid molecule.
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公开(公告)号：US12012437B2
公开(公告)日：2024-06-18
申请号：US17484324
申请日：2021-09-24
Applicant: The Regents of the University of California
Inventor： Stephanie Cherqui
IPC: C07K14/47 , A01K67/0276 , A01K67/0278 , A61K35/28 , A61P25/28 , C12N9/22 , C12N15/11 , C12N15/86
CPC classification number: C07K14/47 , A01K67/0276 , A01K67/0278 , A61K35/28 , A61P25/28 , C12N9/22 , C12N15/11 , C12N15/86 , A01K2207/15 , A01K2217/05 , A01K2217/075 , A01K2217/15 , A01K2227/105 , A01K2267/0318 , C07K2319/60 , C12N2310/20 , C12N2740/16043 , C12N2800/80
Abstract: Provided herein are methods for treating a disease or disorder associated with mitochondrial dysfunction through ex vivo introduction of a nucleic acid molecule into hematopoietic stem and progenitor cells (HSPCs) followed by transplantation of the HSPCs into a subject in need of treatment. The nucleic acid molecule may include a functional human frataxin (hFXN) or may include a gene editing system that when transfected into the cells removes a trinucleotide extension mutation of endogenous hFXN.
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Patent Agency Ranking
公开(公告)号：US12012436B2
公开(公告)日：2024-06-18
申请号：US16082487
申请日：2017-03-15
Applicant: The Regents of the University of California
Inventor： Stephanie Cherqui
IPC: C07K14/47 , A01K67/0276 , A01K67/0278 , A61K35/28 , A61P25/28 , C12N9/22 , C12N15/11 , C12N15/86
CPC classification number: C07K14/47 , A01K67/0276 , A01K67/0278 , A61K35/28 , A61P25/28 , C12N9/22 , C12N15/11 , C12N15/86 , A01K2207/15 , A01K2217/05 , A01K2217/075 , A01K2217/15 , A01K2227/105 , A01K2267/0318 , C07K2319/60 , C12N2310/20 , C12N2740/16043 , C12N2800/80
Abstract: Provided herein are methods for treating a disease or disorder associated with mitochondrial dysfunction through ex vivo introduction of a nucleic acid molecule into hematopoietic stem and progenitor cells (HSPCs) followed by transplantation of the HSPCs into a subject in need of treatment. The nucleic acid molecule may include a functional human frataxin (hFXN) or may include a gene editing system that when transfected into the cells removes a trinucleotide extension mutation of endogenous hFXN.
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公开(公告)号：US11806367B2
公开(公告)日：2023-11-07
申请号：US16493573
申请日：2018-03-15
Applicant: The Regents of the University of California
Inventor： Stephanie Cherqui , Eric Adler , Sylvia Evans
CPC classification number: A61K35/28 , A61K35/14 , A61P3/00 , C07K14/705 , C12N15/86 , A61K38/00 , A61K48/00 , C12N2740/15043
Abstract: Provided herein are methods for treating a lysosomal transmembrane protein disease or disorder through ex vivo introduction of a nucleic acid molecule into hematopoietic stem and progenitor cells (HSPCs) followed by transplantation of the HSPCs into a subject in need of treatment. Also provided are vectors containing the nucleic acid molecule.
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公开(公告)号：US20240293578A1
公开(公告)日：2024-09-05
申请号：US18658077
申请日：2024-05-08
Applicant: The Regents of the University of California
Inventor： Stephanie Cherqui
CPC classification number: A61K48/005 , A61K35/14 , A61K35/28 , C12N9/22 , C12N15/111 , C12N15/85 , C12N2310/20 , C12N2800/80
Abstract: Provided herein are methods for treating a disease or disorder associated with mitochondrial dysfunction through ex vivo introduction of a nucleic acid molecule into hematopoietic stem and progenitor cells (HSPCs) followed by transplantation of the HSPCs into a subject in need of treatment. The nucleic acid molecule may include a functional human frataxin (hFXN) or may include a gene editing system that when transfected into the cells removes a trinucleotide extension mutation of endogenous hFXN.
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公开(公告)号：US12011488B2
公开(公告)日：2024-06-18
申请号：US16820368
申请日：2020-03-16
Applicant: The Regents of the University of California
Inventor： Stephanie Cherqui
CPC classification number: A61K48/005 , A61K35/14 , A61K35/28 , C12N9/22 , C12N15/111 , C12N15/85 , C12N2310/20 , C12N2800/80
Abstract: Provided herein are methods for treating a disease or disorder associated with mitochondrial dysfunction through ex vivo introduction of a nucleic acid molecule into hematopoietic stem and progenitor cells (HSPCs) followed by transplantation of the HSPCs into a subject in need of treatment. The nucleic acid molecule may include a functional human frataxin (hFXN) or may include a gene editing system that when transfected into the cells removes a trinucleotide extension mutation of endogenous hFXN.
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公开(公告)号：US20220009977A1
公开(公告)日：2022-01-13
申请号：US17484324
申请日：2021-09-24
Applicant: The Regents of the University of California
Inventor： Stephanie Cherqui
Abstract: Provided herein are methods for treating a disease or disorder associated with mitochondrial dysfunction through ex vivo introduction of a nucleic acid molecule into hematopoietic stem and progenitor cells (HSPCs) followed by transplantation of the HSPCs into a subject in need of treatment. The nucleic acid molecule may include a functional human frataxin (hFXN) or may include a gene editing system that when transfected into the cells removes a trinucleotide extension mutation of endogenous hFXN.
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