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1.
公开(公告)号:US20220096659A1
公开(公告)日:2022-03-31
申请号:US17508714
申请日:2021-10-22
发明人: Kanut Laoharawee , Kelly M. Podetz-Pedersen , Karen Kozarsky , R. Scott McIvor , Lalitha R. Belur
摘要: A method to prevent, inhibit or treat one or more neurological symptoms associated with a central nervous system disorder, e.g. MPSI or MPSII by, for example, intrathecally, intracerebroventricularly or intravenously administering a rAAV encoding a gene product associated with the disease, e.g., administering to an adult mammal in which the gene product is absent, defective or present at a reduced level relative to a mammal without the disease.
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公开(公告)号:US20180289839A1
公开(公告)日:2018-10-11
申请号:US15574432
申请日:2016-05-13
摘要: A method to prevent, inhibit or treat one or more symptoms associated with disease of the central nervous system by intranasally, intrathecally, intracerebrovascularly or intravenously administering a rAAV encoding a gene product associated with the disease, e.g., a mammal in which the gene product is absent or present at a reduced level relative to a mammal without the disease, in an amount effective, e.g., to provide for cross-correction.
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公开(公告)号:US20180271955A1
公开(公告)日:2018-09-27
申请号:US15989405
申请日:2018-05-25
CPC分类号: A61K38/47 , A61K48/00 , C12N7/00 , C12N15/86 , C12N2750/14143 , C12N2750/14171 , C12N2830/007 , C12Y302/01076 , H05K999/99
摘要: A method to prevent, inhibit or treat one or more symptoms associated with a disease of the central nervous system by intrathecally, intracerebroventricularly or endovascularly administering a rAAV encoding a gene product associated with the disease, e.g., a mammal in which the gene product is absent or present at a reduced level relative to a mammal without the disease.
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公开(公告)号:US20180264090A1
公开(公告)日:2018-09-20
申请号:US15987490
申请日:2018-05-23
CPC分类号: A61K38/47 , A61K48/00 , C12N7/00 , C12N15/86 , C12N2750/14143 , C12N2750/14171 , C12N2830/007 , C12Y302/01076 , H05K999/99
摘要: A method to prevent, inhibit or treat one or more symptoms associated with a disease of the central nervous system by intrathecally, intracerebroventricularly or endovascularly administering a rAAV encoding a gene product associated with the disease, e.g., a mammal in which the gene product is absent or present at a reduced level relative to a mammal without the disease.
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5.
公开(公告)号:US20220211875A1
公开(公告)日:2022-07-07
申请号:US17574118
申请日:2022-01-12
申请人: Regents of the University of Minnesota , HealthPartners Institute f/k/a HealthPartners Research Foundation , Regenxbio Inc.
IPC分类号: A61K48/00 , A61K31/675 , A61K38/47 , A61K45/06 , C12N15/86 , A61P25/28 , A61K9/00 , C12N7/00
摘要: A method to prevent, inhibit or treat one or more symptoms associated with disease of the central nervous system by intranasally, intrathecally, intracerebrovascularly or intravenously
administering a rAAV encoding a gene product associated with the disease, e.g., a mammal in which the gene product is absent or present at a reduced level relative to a mammal without the disease, in an amount effective, e.g., to provide for cross-correction.-
公开(公告)号:US20210346473A1
公开(公告)日:2021-11-11
申请号:US17212516
申请日:2021-03-25
摘要: A method to prevent, inhibit or treat one or more symptoms associated with a disease of the central nervous system by intrathecally, intracerebroventricularly or endovascularly administering a rAAV encoding a gene product associated with the disease, e.g., a mammal in which the gene product is absent or present at a reduced level relative to a mammal without the disease.
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公开(公告)号:US20210085759A1
公开(公告)日:2021-03-25
申请号:US17118353
申请日:2020-12-10
摘要: A method to prevent, inhibit or treat one or more symptoms associated with a disease of the central nervous system by intrathecally, intracerebroventricularly or endovascularly administering a rAAV encoding a gene product associated with the disease, e.g., a mammal in which the gene product is absent or present at a reduced level relative to a mammal without the disease.
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公开(公告)号:US20190298812A1
公开(公告)日:2019-10-03
申请号:US16438143
申请日:2019-06-11
摘要: A method to prevent, inhibit or treat one or more symptoms associated with a disease of the central nervous system by intrathecally, intracerebroventricularly or endovascularly administering a rAAV encoding a gene product associated with the disease, e.g., a mammal in which the gene product is absent or present at a reduced level relative to a mammal without the disease.
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公开(公告)号:US20180271957A1
公开(公告)日:2018-09-27
申请号:US15995055
申请日:2018-05-31
CPC分类号: A61K38/47 , A61K48/00 , C12N7/00 , C12N15/86 , C12N2750/14143 , C12N2750/14171 , C12N2830/007 , C12Y302/01076 , H05K999/99
摘要: A method to prevent, inhibit or treat one or more symptoms associated with a disease of the central nervous system by intrathecally, intracerebroventricularly or endovascularly administering a rAAV encoding a gene product associated with the disease, e.g., a mammal in which the gene product is absent or present at a reduced level relative to a mammal without the disease.
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公开(公告)号:US20180271956A1
公开(公告)日:2018-09-27
申请号:US15995045
申请日:2018-05-31
摘要: A method to prevent, inhibit or treat one or more symptoms associated with a disease of the central nervous system by intrathecally, intracerebroventricularly or endovascularly administering a rAAV encoding a gene product associated with the disease, e.g., a mammal in which the gene product is absent or present at a reduced level relative to a mammal without the disease.
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