公开(公告)号：US09193969B2

公开(公告)日：2015-11-24

申请号：US13066590

申请日：2011-04-19

申请人： Andrés Pablo Montefeltro ,  Gabriel Alvarado Urbina ,  Analia Bortolozzi Biassoni ,  Francesc Artigas Pérez ,  Miquel Vila Bover ,  Maria del Carmen Carmona Orozco

发明人： Andrés Pablo Montefeltro ,  Gabriel Alvarado Urbina ,  Analia Bortolozzi Biassoni ,  Francesc Artigas Pérez ,  Miquel Vila Bover ,  Maria del Carmen Carmona Orozco

IPC分类号： C07H21/04 ,  C12N15/11 ,  A61K47/48 ,  A61K49/00 ,  C12N15/113 ,  A61K48/00

CPC分类号： A61K47/48023 ,  A61K31/135 ,  A61K31/472 ,  A61K31/7105 ,  A61K31/713 ,  A61K47/543 ,  A61K47/549 ,  A61K47/55 ,  A61K47/60 ,  A61K49/0002 ,  C07D207/46 ,  C07D217/04 ,  C12N15/111 ,  C12N15/1138 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/321 ,  C12N2310/341 ,  C12N2310/351 ,  C12N2310/3515 ,  C12N2320/32 ,  C12N2310/3521

摘要： The invention provides a conjugate comprising (i) a nucleic acid which is complementary to a target nucleic acid sequence and which expression prevents or reduces expression of the target nucleic acid and (ii) a selectivity agent which is capable of binding with high affinity to a neurotransmitter transporter. The conjugates of the present invention are useful for the delivery of the nucleic acid to a cell of interests and thus, for the treatment of diseases which require a down-regulation of the protein encoded by the target nucleic acid as well as for the delivery of imaging agents to the cells for diagnostic purposes.

摘要翻译： 本发明提供了一种缀合物，其包含（i）与靶核酸序列互补的核酸，并且该表达阻止或减少靶核酸的表达，和（ii）能够以高亲和力结合的选择剂 神经递质转运蛋白 本发明的缀合物可用于将核酸递送至感兴趣的细胞，因此用于治疗需要下调靶核酸编码的蛋白质以及用于递送 成像剂到细胞用于诊断目的。

公开(公告)号：US20120322991A1

公开(公告)日：2012-12-20

申请号：US13066590

申请日：2011-04-19

申请人： Andres Pablo Montefeltro ,  Gabriel Alvarado Urbina ,  Analia Bortolozzi Biassoni ,  Francesc Artigas Perez ,  Miquel Vila Bover

发明人： Andres Pablo Montefeltro ,  Gabriel Alvarado Urbina ,  Analia Bortolozzi Biassoni ,  Francesc Artigas Perez ,  Miquel Vila Bover

IPC分类号： C07H21/00

CPC分类号： A61K47/48023 ,  A61K31/135 ,  A61K31/472 ,  A61K31/7105 ,  A61K31/713 ,  A61K47/543 ,  A61K47/549 ,  A61K47/55 ,  A61K47/60 ,  A61K49/0002 ,  C07D207/46 ,  C07D217/04 ,  C12N15/111 ,  C12N15/1138 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/321 ,  C12N2310/341 ,  C12N2310/351 ,  C12N2310/3515 ,  C12N2320/32 ,  C12N2310/3521

摘要： The invention provides a conjugate comprising (i) a nucleic acid which is complementary to a target nucleic acid sequence and which expression prevents or reduces expression of the target nucleic acid and (ii) a selectivity agent which is capable of binding with high affinity to a neurotransmitter transporter. The conjugates of the present invention are useful for the delivery of the nucleic acid to a cell of interests and thus, for the treatment of diseases which require a down-regulation of the protein encoded by the target nucleic acid as well as for the delivery of imaging agents to the cells for diagnostic purposes.

摘要翻译： 本发明提供了一种缀合物，其包含（i）与靶核酸序列互补的核酸，并且该表达阻止或减少靶核酸的表达，和（ii）能够以高亲和力结合的选择剂 神经递质转运蛋白 本发明的缀合物可用于将核酸递送至感兴趣的细胞，因此用于治疗需要下调靶核酸编码的蛋白质以及用于递送 成像剂到细胞用于诊断目的。

公开(公告)号：US5700677A

公开(公告)日：1997-12-23

申请号：US799769

申请日：1991-11-27

申请人： Roberto Crea ,  Roy Hoi Loi Pang ,  Hermann Oppermann ,  Peter C. Keck ,  Gabriel Alvarado-Urbina ,  Gay-May Wu ,  Charles M. Cohen

发明人： Roberto Crea ,  Roy Hoi Loi Pang ,  Hermann Oppermann ,  Peter C. Keck ,  Gabriel Alvarado-Urbina ,  Gay-May Wu ,  Charles M. Cohen

IPC分类号： C12N9/64 ,  A61K38/00 ,  A61K38/46 ,  A61P7/02 ,  C07K14/00 ,  C07K14/31 ,  C12N1/20 ,  C12N9/72 ,  C12N15/00 ,  C12N15/09 ,  C12P21/02 ,  C12R1/19 ,  A61K38/49 ,  C07K14/745

CPC分类号： C07K14/31 ,  C12N9/6459 ,  C12Y304/21069 ,  A61K38/00

摘要： Protein analogues of tissue plasminogen activator (tPA) are described. The analogues exhibit the proteolytic function of natural tPA and optionally fibrin binding activity, but are molecules, generally of lower molecular weight than natural tPA, designed for efficient expression in prokaryotic host cell systems. The analogues can comprise a catalytic fragment of tPA or a catalytic fragment of tPA linked to a polypeptide which stabilizes the catalytic fragment, provides for efficient expression of the fragment or confers a fibrin binding capability. Fibrin binding polypeptides can be a polypeptide fragments derived from tPA which embody the fibrin binding domain(s) of natural tPA or they can be an exogenous (non-tPA) polypeptides of eukaryotic or prokaryotic origin which exhibit fibrin binding affinity such as the antigen binding fragment of an antifibrin immunoglobulin or the B domain of protein A. Genetic constructs for expression of the analogues are also provided.

摘要翻译： 描述了组织纤溶酶原激活物（tPA）的蛋白质类似物。 类似物表现出天然tPA和任选的纤维蛋白结合活性的蛋白水解功能，但是通常具有比天然tPA低的分子量的分子，其设计用于在原核宿主细胞系统中有效表达。 类似物可以包含tPA的催化片段或与稳定催化片段的多肽连接的tPA的催化片段，提供片段的有效表达或赋予纤维蛋白结合能力。 纤维蛋白结合多肽可以是衍生自tPA的多肽片段，其表现为天然tPA的纤维蛋白结合结构域，或者它们可以是表现出纤维蛋白结合亲和力的真核或原核来源的外源（非tPA）多肽，例如抗原结合 抗纤维蛋白免疫球蛋白的片段或蛋白质B的B结构域。还提供了用于表达类似物的遗传构建体。