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公开(公告)号：US08580922B2
公开(公告)日：2013-11-12
申请号：US13411287
申请日：2012-03-02
申请人： Paolo Martini , Michael Concino
发明人： Paolo Martini , Michael Concino
CPC分类号： C12N9/2402 , A61K9/0019 , A61K38/30 , A61K38/465 , A61K38/47 , A61K47/48338 , A61K47/65 , C07K7/08 , C07K14/65 , C07K2319/01 , C12Y302/0105
摘要： Disclosed herein are novel peptide linkers and polypeptide compositions comprising the linkers (e.g., chimeric polypeptides) and methods of using the polypeptide compositions. The compositions and methods are particularly useful for targeting/delivering a polypeptide or protein of interest (e.g., a therapeutic polypeptide) to a cell, tissue or organ of interest in order to treat various diseases or disorders (e.g., lysosomal storage disorders).
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公开(公告)号：US20120232021A1
公开(公告)日：2012-09-13
申请号：US13411287
申请日：2012-03-02
申请人： Paolo Martini , Michael Concino
发明人： Paolo Martini , Michael Concino
CPC分类号： C12N9/2402 , A61K9/0019 , A61K38/30 , A61K38/465 , A61K38/47 , A61K47/48338 , A61K47/65 , C07K7/08 , C07K14/65 , C07K2319/01 , C12Y302/0105
摘要： Disclosed herein are novel peptide linkers and polypeptide compositions comprising the linkers (e.g., chimeric polypeptides) and methods of using the polypeptide compositions. The compositions and methods are particularly useful for targeting/delivering a polypeptide or protein of interest (e.g., a therapeutic polypeptide) to a cell, tissue or organ of interest in order to treat various diseases or disorders (e.g., lysosomal storage disorders).
摘要翻译： 本文公开了包含接头（例如嵌合多肽）的新型肽接头和多肽组合物以及使用该多肽组合物的方法。 为了治疗各种疾病或病症（例如溶酶体贮积障碍），组合物和方法特别可用于靶向/递送感兴趣的多肽或蛋白质（例如，治疗性多肽）至感兴趣的细胞，组织或器官。
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公开(公告)号：US09260495B2
公开(公告)日：2016-02-16
申请号：US14127074
申请日：2012-06-15
CPC分类号： C07K14/47 , A01K2217/075 , A01K2227/105 , A01K2267/0306 , A61K38/00 , A61K38/1709 , A61K47/645 , A61K49/0008 , C07K7/08 , C07K2319/07 , C07K2319/10
摘要： The present invention provides, among other things, compositions and methods for treatment of Friedrich's Ataxia based on effective targeting of a therapeutic moiety to mitochondria that can substitute for natural FXN protein activity or rescue one or more phenotypes or symptoms associated with frataxin-deficiency. In some embodiments, the present invention provides a targeted therapeutic comprising a therapeutic moiety, which is a polypeptide having an N-terminus and a C-terminus, a mitochondrial targeting sequence associated with the therapeutic moiety at the N-terminus, and a mitochondrial membrane-penetrating peptide associated with the therapeutic moiety at the C-terminus, wherein the therapeutic moiety is targeted to mitochondria upon cellular entry.
摘要翻译： 本发明尤其提供了用于治疗弗里德里希共济失调的组合物和方法，其基于可以替代天然FXN蛋白活性或拯救一种或多种与frataxin缺陷相关的表型或症状的线粒体的治疗性部分的有效靶向。 在一些实施方案中，本发明提供靶向治疗剂，其包含治疗部分，其为具有N末端和C末端的多肽，与N末端的治疗部分相关的线粒体靶向序列，以及线粒体膜 与C-末端的治疗部分相关的穿透肽，其中治疗部分在细胞进入时靶向线粒体。
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公开(公告)号：US20140135275A1
公开(公告)日：2014-05-15
申请号：US14127074
申请日：2012-06-15
CPC分类号： C07K14/47 , A01K2217/075 , A01K2227/105 , A01K2267/0306 , A61K38/00 , A61K38/1709 , A61K47/645 , A61K49/0008 , C07K7/08 , C07K2319/07 , C07K2319/10
摘要： The present invention provides, among other things, compositions and methods for treatment of Friedrich's Ataxia based on effective targeting of a therapeutic moiety to mitochondria that can substitute for natural FXN protein activity or rescue one or more phenotypes or symptoms associated with frataxin-deficiency. In some embodiments, the present invention provides a targeted therapeutic comprising a therapeutic moiety, which is a polypeptide having an N-terminus and a C-terminus, a mitochondrial targeting sequence associated with the therapeutic moiety at the N-terminus, and a mitochondrial membrane-penetrating peptide associated with the therapeutic moiety at the C-terminus, wherein the therapeutic moiety is targeted to mitochondria upon cellular entry.
摘要翻译： 本发明尤其提供了用于治疗弗里德里希共济失调的组合物和方法，其基于可以替代天然FXN蛋白活性或拯救一种或多种与frataxin缺陷相关的表型或症状的线粒体的治疗性部分的有效靶向。 在一些实施方案中，本发明提供靶向治疗剂，其包含治疗部分，其为具有N末端和C末端的多肽，与N末端的治疗部分相关的线粒体靶向序列，以及线粒体膜 与C-末端的治疗部分相关的穿透肽，其中治疗部分在细胞进入时靶向线粒体。
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