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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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3.发明公开公开(公告)号：US20240316217A1
公开(公告)日：2024-09-26
申请号：US18264364
申请日：2022-02-04
发明人： R. Scott McIvor , Nicholas Alexander Piers Sascha Buss , Troy C. Lund , Elizabeth Braunlin , Lalitha R. Belur , Carolyn Fairbanks , Marie-Laure Nevoret
CPC分类号： A61K48/005 , A61K9/0019 , A61K9/0085 , A61K38/47 , A61K48/0075 , A61K48/0083 , A61P3/06 , A61P9/00 , A61P19/00 , C12N15/86 , C12Y302/01076 , C12N2750/14143
摘要： A method to prevent, inhibit the progression of, reduce the severity of, or treat cardiac, vascular or skeletal dysfunction or defect(s) in a human having lysosomal storage disorder, is provided.
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4.发明申请公开(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：US20230226225A1
公开(公告)日：2023-07-20
申请号：US18150740
申请日：2023-01-05
IPC分类号： A61K48/00 , A61K31/675 , A61K38/47 , A61K45/06 , C12N15/86 , A61P25/28 , A61K9/00 , C12N7/00
CPC分类号： A61K48/0075 , A61K48/0083 , A61K31/675 , A61K38/47 , A61K45/06 , C12N15/86 , C12Y302/01076 , A61P25/28 , A61K9/0043 , C12N7/00 , C12N2750/14143 , C12N2750/14171
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：US20180071373A1
公开(公告)日：2018-03-15
申请号：US15813908
申请日：2017-11-15
CPC分类号： A61K48/0075 , A61K9/0043 , A61K31/675 , A61K38/47 , A61K45/06 , A61K48/0083 , A61P25/28 , C12N7/00 , C12N15/86 , C12N2750/14143 , C12N2750/14171 , C12Y302/01076 , A61K2300/00
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.
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公开(公告)号：US09827295B2
公开(公告)日：2017-11-28
申请号：US14889750
申请日：2014-05-15
IPC分类号： A61K48/00 , C12N15/86 , C12N15/11 , C12N15/113 , C12N15/00 , C12N15/09 , C12N15/10 , A61K38/47 , C12N7/00
CPC分类号： A61K38/47 , A61K48/00 , C12N7/00 , C12N15/86 , C12N2750/14143 , C12N2750/14171 , C12N2830/007 , C12Y302/01076
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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9.发明申请ADENO-ASSOCIATED VIRUS MEDIATED GENE TRANSFER TO THE CENTRAL NERVOUS SYSTEM 有权ADENO相关病毒介导的基因转移到中枢神经系统公开(公告)号：US20160120960A1
公开(公告)日：2016-05-05
申请号：US14889750
申请日：2014-05-15
CPC分类号： A61K38/47 , A61K48/00 , C12N7/00 , C12N15/86 , C12N2750/14143 , C12N2750/14171 , C12N2830/007 , C12Y302/01076
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.
摘要翻译： 通过鞘内，脑室内或血管内施用编码与该疾病相关的基因产物的rAAV，例如基因产物不存在的哺乳动物来预防，抑制或治疗与中枢神经系统疾病相关的一种或多种症状的方法 或相对于没有疾病的哺乳动物的水平降低。
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10.发明申请公开(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.
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