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公开(公告)号：US10472650B2
公开(公告)日：2019-11-12
申请号：US15551869
申请日：2016-02-22
IPC分类号： C12N7/00 , C12N15/00 , A61K39/23 , C12N15/861 , C12N15/86 , A61P27/00 , A61K48/00 , C07K14/005
摘要： The present disclosure provides targeting peptides and vectors containing a sequence that encodes targetting peptides that deliver agents, to the eye. The present inventors have discovered peptides that function to target agents, such as viral vectors, to ocular cells. The present disclosure describes a method to utilize these novel peptides to direct, for example, viral capsids to the cell type of interest. In this instance, ocular cells (such as retinal cells) are targeted by the identified peptides. Vectors harboring capsid proteins modified to include such peptides can be used to provide therapeutic agents to the eye.
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公开(公告)号：US08691948B2
公开(公告)日：2014-04-08
申请号：US13652177
申请日：2012-10-15
CPC分类号： A61K38/162 , A61K48/00 , C07K5/08 , C07K5/10 , C07K7/06 , C07K14/005 , C07K2319/33 , C12N15/86 , C12N2750/14133 , C12N2750/14143 , C12N2750/14145 , C12N2810/40 , G01N33/5058
摘要： The present disclosure provides targeting peptides and vectors containing a sequence that encodes targeting peptides that deliver agents to the brain.
摘要翻译： 本公开提供了靶向肽和载体，其含有编码将药物递送至脑的靶向肽的序列。
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公开(公告)号：US10391184B2
公开(公告)日：2019-08-27
申请号：US14907776
申请日：2014-07-20
IPC分类号： A61K48/00 , A61K31/343
摘要： The present disclosure provides methods of treating a disease or delivering a therapeutic agent to a mammal comprising administering to the mammal's cisterna magna and/or ventricle an rAAV particle containing a vector comprising a nucleic acid encoding a therapeutic protein inserted between a pair of AAV inverted terminal repeats in a manner such that cells with access to the cerebrospinal fluid (CSF) express the therapeutic agent and in certain embodiments secretes the therapeutic agent into the CSF for distribution to the brain.
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公开(公告)号：US20180236105A1
公开(公告)日：2018-08-23
申请号：US15769931
申请日：2016-10-24
CPC分类号： A61K48/0075 , A61K9/0085 , A61K31/5377 , A61K38/13 , A61K38/4813 , A61K48/005 , A61P25/28 , C07K14/015 , C12N7/00 , C12N9/485 , C12N2750/14143 , C12N2750/14171 , C12Y304/14009 , A61K2300/00
摘要： The present disclosure provides methods of treating a lysosomal storage disorder in a mammal which method comprises administering AAV particles encoding a polypeptide directly to the central nervous system of the mammal in conjunction with administering at least two immunosuppressive agents. AAV particles may be delivered by direct injection into the brain, spinal cord, cerebral spinal fluid or a portion thereof.
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公开(公告)号：US20170348386A1
公开(公告)日：2017-12-07
申请号：US15407121
申请日：2017-01-16
CPC分类号： A61K38/162 , A61K48/00 , C07K5/08 , C07K5/10 , C07K7/06 , C07K14/005 , C07K2319/33 , C12N15/86 , C12N2750/14133 , C12N2750/14143 , C12N2750/14145 , C12N2810/40 , G01N33/5058
摘要： The present disclosure provides targeting peptides and vectors containing a sequence that encodes targeting peptides that deliver agents to the brain.
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公开(公告)号：US20130142764A1
公开(公告)日：2013-06-06
申请号：US13652177
申请日：2012-10-15
CPC分类号： A61K38/162 , A61K48/00 , C07K5/08 , C07K5/10 , C07K7/06 , C07K14/005 , C07K2319/33 , C12N15/86 , C12N2750/14133 , C12N2750/14143 , C12N2750/14145 , C12N2810/40 , G01N33/5058
摘要： The present disclosure provides targeting peptides and vectors containing a sequence that encodes targeting peptides that deliver agents to the brain.
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公开(公告)号：US20160166709A1
公开(公告)日：2016-06-16
申请号：US14907776
申请日：2014-07-20
IPC分类号： A61K48/00 , A61K31/343
CPC分类号： A61K48/0058 , A61K31/343 , A61K48/00 , A61K48/0075 , C12N2750/14143
摘要： The present disclosure provides methods of treating a disease or delivering a therapeutic agent to a mammal comprising administering to the mammal's cisterna magna and/or ventricle an rAAV particle containing a vector comprising a nucleic acid encoding a therapeutic protein inserted between a pair of AAV inverted terminal repeats in a manner such that cells with access to the cerebrospinal fluid (CSF) express the therapeutic agent and in certain embodiments secretes the therapeutic agent into the CSF for distribution to the brain.
摘要翻译： 本公开提供了治疗疾病或向哺乳动物递送治疗剂的方法，包括向哺乳动物的大池和/或心室施用含有载体的rAAV颗粒，所述载体包含插入在一对AAV倒置末端之间的编码治疗性蛋白质的核酸 以使得接触到脑脊液（CSF）的细胞表达治疗剂的方式重复，并且在某些实施方案中将治疗剂分泌到CSF中以分配给脑。
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公开(公告)号：US20150374781A1
公开(公告)日：2015-12-31
申请号：US14679542
申请日：2015-04-06
IPC分类号： A61K38/16 , G01N33/50 , C07K14/005
CPC分类号： A61K38/162 , A61K48/00 , C07K5/08 , C07K5/10 , C07K7/06 , C07K14/005 , C07K2319/33 , C12N15/86 , C12N2750/14133 , C12N2750/14143 , C12N2750/14145 , C12N2810/40 , G01N33/5058
摘要： The present disclosure provides targeting peptides and vectors containing a sequence that encodes targeting peptides that deliver agents to the brain.
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公开(公告)号：US20140161777A1
公开(公告)日：2014-06-12
申请号：US14180129
申请日：2014-02-13
CPC分类号： A61K38/162 , A61K48/00 , C07K5/08 , C07K5/10 , C07K7/06 , C07K14/005 , C07K2319/33 , C12N15/86 , C12N2750/14133 , C12N2750/14143 , C12N2750/14145 , C12N2810/40 , G01N33/5058
摘要： The present disclosure provides targeting peptides and vectors containing a sequence that encodes targeting peptides that deliver agents to the brain.
摘要翻译： 本公开提供了靶向肽和载体，其含有编码将药物递送至脑的靶向肽的序列。
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