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71. 发明申请

WO2017136500A1 GENE THERAPY FOR TREATING MUCOPOLYSACCHARIDOSIS TYPE I 审中-公开
标题翻译：基因疗法治疗粘多糖1型
公开(公告)号：WO2017136500A1
公开(公告)日：2017-08-10
申请号：PCT/US2017/016133
申请日：2017-02-02
申请人： THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA
发明人： HINDERER, Christian , WILSON, James, M.
IPC分类号： C12N15/86 , A61K48/00 , C12N9/24 , A61K39/00
CPC分类号： C12N15/86 , A61K48/005 , A61K48/0075 , C12N9/2402 , C12N2750/14143 , C12N2830/15 , C12Y302/01076
摘要： A suspension useful for AAV9-mediated intrathecal/intracisternal and/or systemic delivery of an expression cassette containing a hIDUA gene is provided herein. Also provided are methods and kits containing these vectors and compositions useful for treating MPSI and the symptoms associated with Hurler, Hurler-Scheie and Scheie syndromes.
摘要翻译：本文提供了用于AAV9介导的鞘内/脑池内和/或全身递送含有hIDUA基因的表达盒的悬液。还提供了包含这些载体和组合物的方法和试剂盒，用于治疗MPSI以及与Hurler，Hurler-Scheie和Scheie综合征有关的症状。

72. 发明申请

WO2016037138A8 GLOBIN GENE THERAPY FOR TREATING HEMOGLOBINOPATHIES 审中-公开
标题翻译：用于治疗肝细胞病变的GLOBIN基因治疗
公开(公告)号：WO2016037138A8
公开(公告)日：2017-03-16
申请号：PCT/US2015048698
申请日：2015-09-04
申请人： MEMORIAL SLOAN-KETTERING CANCER CENTER , UNIV WASHINGTON
发明人： SADELAIN MICHEL , RIVIERE ISABELLE , MANSILLA-SOTO JORGE , WANG XIUYAN , STAMATOYANNOPOULOS GEORGE , STAMATOYANNOPOULOS JOHN , LIU MINGDONG
IPC分类号： C12N15/113
CPC分类号： A61K48/0066 , A61K48/0058 , C07K14/805 , C12N9/22 , C12N15/85 , C12N15/86 , C12N2740/10043 , C12N2740/15043 , C12N2740/16043 , C12N2830/008 , C12N2830/15 , C12N2830/30 , C12N2830/40 , C12N2830/46 , C12N2830/48 , C12N2830/50
摘要： The presently disclosed subject matter provides for expression cassettes that allow for expression of a globin gene or a functional portion thereof, vectors comprising thereof, and cells transduced with such expression cassettes and vectors. The presently disclosed subject matter further provides methods for treating a hemoglobinopathy in a subject comprising administering an effective amount of such transduced cells to the subject.
摘要翻译：目前公开的主题提供了允许表达珠蛋白基因或其功能部分的表达载体，其包含的载体，以及用这种表达盒和载体转导的细胞。目前公开的主题还提供了治疗受试者的血红蛋白病的方法，包括向受试者施用有效量的这种转导的细胞。

73. 发明申请

WO2016133917A1 RAAV VECTOR COMPOSITIONS, METHODS FOR TARGETING VASCULAR ENDOTHELIAL CELLS AND USE IN TREATMENT OF TYPE I DIABETES 审中-公开
标题翻译： RAAV VECTOR组合物，用于靶向血管内皮细胞的方法和用于治疗I型糖尿病的治疗
公开(公告)号：WO2016133917A1
公开(公告)日：2016-08-25
申请号：PCT/US2016/018098
申请日：2016-02-16
申请人： UNIVERSITY OF FLORIDA RESEARCH FOUNDATION
发明人： HAUSWIRTH, William W. , BOYE, Sanford L. , LIPINSKI, Daniel M. , BOULTON, Michael E. , BOYE, Shannon
IPC分类号： C12N15/86 , A61K35/76 , C12N9/78 , A61K38/50
CPC分类号： C12N15/86 , A61K38/00 , A61K38/50 , A61K48/0058 , C07K14/005 , C07K14/705 , C12N7/00 , C12N9/78 , C12N2750/14121 , C12N2750/14122 , C12N2750/14143 , C12N2830/008 , C12N2830/15 , C12N2830/85 , C12Y305/04016
摘要： Disclosed are capsid-modified rAAV expression vectors, as well as infectious virions, compositions, and pharmaceutical formulations that include them. Also disclosed are methods of preparing and using novel capsid-protein-mutated rAAV vector constructs in a variety of diagnostic and therapeutic applications including, inter alia , as delivery agents for diagnosis, treatment, or amelioration of one or more diseases, disorders, or dysfunctions of the mammalian vascular system, and complications from Type I diabetes. Also disclosed are methods for systemic and tissue-localized delivery of therapeutic rAAV-based gene expression cassettes to vascular endothelial cells, tissues, and organs, as well as use of the disclosed compositions in the manufacture of medicaments for a variety of in vitro and/or in vivo applications including the treatment of vasculitis, and complications arising from Type I diabetes, such as macular edema, nephropathy, diabetic retinopathy, and the like.
摘要翻译：公开了衣壳修饰的rAAV表达载体，以及感染性病毒粒子，组合物和包括它们的药物制剂。还公开了在各种诊断和治疗应用中制备和使用新的衣壳蛋白突变的rAAV载体构建体的方法，包括特别地，作为用于诊断，治疗或改善一种或多种疾病，障碍或功能障碍的递送剂的哺乳动物血管系统，以及I型糖尿病的并发症。还公开了将治疗性基于rAAV的基因表达盒全身和组织定位递送至血管内皮细胞，组织和器官的方法，以及所公开的组合物在制备用于各种体外和/ 或体内应用，包括治疗血管炎，以及I型糖尿病引起的并发症，例如黄斑水肿，肾病，糖尿病性视网膜病变等。

74. 发明申请

WO2013061010A1 UNITES DE TRANSCRIPTION ET LEUR UTILISATION DANS DES VECTEURS D'EXPRESSION 审中-公开
标题翻译：（YB2 / 0）表达载体的转录单位及其用途
公开(公告)号：WO2013061010A1
公开(公告)日：2013-05-02
申请号：PCT/FR2012/052496
申请日：2012-10-29
申请人： LFB BIOTECHNOLOGIES , FONTAYNE, Alexandre , COUTARD, François
发明人： FONTAYNE, Alexandre , COUTARD, François
IPC分类号： C12N15/85 , C07K16/00
CPC分类号： C12N15/85 , C07K16/2869 , C07K16/2887 , C07K16/2896 , C12N2830/15 , C12N2830/42 , C12P21/00
摘要： La présente invention concerne une unité de transcription constituée d'un polynucléotide comprenant l'enhancer du virus hCMVe, ledit enhancer ayant la séquence nucléotidique SEQ ID NO: 1, ou un acide nucléotidique présentant au moins 70% d'identité de séquence avec la séquence SEQ IDNO : 1 et possédant essentiellement des propriétés d'activation de la transcription, et la région promotrice dela cycline dépendante des kinases 9 (CDK9), ladite région promotrice ayant la séquence nucléotidique SEQ ID NO: 2, ou un acide nucléotidique présentant au moins 70% d'identité de séquence avec la séquence SEQ IDNO : 2 et possédant essentiellement une activité promotrice.
摘要翻译：本发明涉及由包含hCMVe病毒增强子的多核苷酸组成的转录单位，其中所述增强子具有核苷酸序列SEQ ID NO：1或与序列SEQ ID NO：1具有至少70％序列同一性的核苷酸并且基本上具有转录激活特性，以及细胞周期蛋白依赖性激酶9（CDK9）的启动子区，其中所述启动子区具有核苷酸序列SEQ ID NO：2，或与序列具有至少70％序列同一性的核苷酸 SEQ ID NO：2并且基本上具有启动子活性。

75. 发明申请

WO2009130208A1 LIVER-SPECIFIC NUCLEIC ACID REGULATORY ELEMENTS AND METHODS AND USE THEREOF 审中-公开
标题翻译：肝特异性核酸调节因子及其使用方法
公开(公告)号：WO2009130208A1
公开(公告)日：2009-10-29
申请号：PCT/EP2009/054724
申请日：2009-04-21
申请人： VIB VZW , LIFE SCIENCES RESEARCH PARTNERS VZW , UNIVERSITEIT GENT , CHUAH, Marinee , VANDENDRIESSCHE, Thierry , DE BLESER, Pieter
发明人： CHUAH, Marinee , VANDENDRIESSCHE, Thierry , DE BLESER, Pieter
IPC分类号： A61K38/36 , A61K48/00 , C07K14/755 , C07K14/81 , C12N15/864
CPC分类号： A61K48/0058 , A61K38/4846 , A61K48/00 , C07K14/8121 , C12N9/644 , C12N15/52 , C12N15/63 , C12N15/85 , C12N15/86 , C12N2750/14143 , C12N2799/025 , C12N2799/027 , C12N2830/008 , C12N2830/15 , C12N2830/85 , C12Y304/21022
摘要： The present invention relates to nucleic acid regulatory elements that are able to enhance liver-specific expression of genes, methods employing these regulatory elements and uses of these elements. Expression cassettes and vectors containing these nucleic acid regulatory elements are also disclosed. The present invention is particularly useful for applications using gene therapy.
摘要翻译：本发明涉及能够增强基因的肝特异性表达的核酸调节元件，采用这些调控元件的方法和这些元件的用途。还公开了含有这些核酸调节元件的表达盒和载体。本发明对于使用基因治疗的应用特别有用。

76. 发明申请

WO2009020236A2 NOVEL VIRAL VECTOR 审中-公开
标题翻译：新的VIRAL VECTOR
公开(公告)号：WO2009020236A2
公开(公告)日：2009-02-12
申请号：PCT/JP2008/064503
申请日：2008-08-06
申请人： EDUCATIONAL FOUNDATION JICHI MEDICAL UNIVERSITY , OTSUKA PHARMACEUTICAL CO., LTD. , YOSHIDA, Shigeto , KAWASAKI, Masanori , MATSUMOTO, Makoto , OHBA, Yoshio , SAITO, Masahiro , GOTO, Yoshihiro , INAGAKI, Katsuya , MIZUKOSHI, Masami , HARIGUCHI, Norimitsu , HIROTA, Kuniko
发明人： YOSHIDA, Shigeto , KAWASAKI, Masanori , MATSUMOTO, Makoto , OHBA, Yoshio , SAITO, Masahiro , GOTO, Yoshihiro , INAGAKI, Katsuya , MIZUKOSHI, Masami , HARIGUCHI, Norimitsu , HIROTA, Kuniko
IPC分类号： C12N15/866 , A61K39/145 , A61K39/015
CPC分类号： C07K16/1018 , A61K39/015 , A61K39/12 , A61K39/145 , A61K2039/5256 , C07K14/005 , C07K14/445 , C07K16/205 , C07K2319/735 , C12N7/00 , C12N15/86 , C12N2710/14122 , C12N2710/14143 , C12N2760/16122 , C12N2760/16134 , C12N2830/15 , C12N2830/60 , C12N2830/85 , Y02A50/412
摘要： The present invention provides a novel transfer vector and a recombinant baculovirus; methods for the production thereof; and pharmaceuticals containing the recombinant baculovirus as an active ingredient, which are useful as preventive or therapeutic drugs for infectious diseases such as malaria and influenza. More specifically, the invention provides a recombinant transfer vector capable of expressing a foreign gene fused to a virus gene under the control of a dual promoter; a recombinant baculovirus; methods for the production thereof; and pharmaceuticals containing the recombinant baculovirus as an active ingredient.
摘要翻译：本发明提供新型转移载体和重组杆状病毒; 生产方法; 以及含有重组杆状病毒作为活性成分的药物，其可用作疟疾和流感等感染性疾病的预防或治疗药物。更具体地，本发明提供能够表达在双重启动子控制下与病毒基因融合的外源基因的重组转移载体; 重组杆状病毒; 生产方法; 以及含有重组杆状病毒作为有效成分的药物。

77. 发明申请

WO2007127428A8 DOUBLE-STRANDED/SELF-COMPLEMENTARY VECTORS WITH A TRUNCATED CBA PROMOTER AND METHODS OF GENE DELIVERY 审中-公开
标题翻译：具有截断CBA促进器的双重条纹/自补偿矢量和基因传递方法
公开(公告)号：WO2007127428A8
公开(公告)日：2008-04-10
申请号：PCT/US2007010338
申请日：2007-04-27
申请人： UNIV FLORIDA , BOYE SANFORD LEON , HAUSWIRTH WILLIAM W , BYRNE BARRY JOHN
发明人： BOYE SANFORD LEON , HAUSWIRTH WILLIAM W , BYRNE BARRY JOHN
IPC分类号： C12N15/00 , A61K38/00 , C12N5/00
CPC分类号： C12N15/86 , C12N2750/14143 , C12N2830/15
摘要： The present inventors concerns vectors carrying a truncated chimeric CMV- chicken ß-actin (smCBA) promoter in which the hybrid chicken ß-actin/rabbit ß-globin intron is greatly shortened, and their use to deliver to an operatively linked polynucleotide to host cells in vitro or in vivo, resulting in expression of the polynucleotide in the host cells. In one embodiment, the vector carrying the smCBA promoter is administered to the eye. In another embodiment, the vector carrying the smCBA promoter is a self- complementary adeno-associated virus (AAV). The AAV vector may be of any serotype (e.g., type 1, type 2, type 3, type 4, type 5, type 6, type 7, type 8, type 9, type 10). In another embodiment, a self-complementary vector carrying the smCBA promoter is administered to the eye. Another aspect of the invention concerns host cells carrying a vector of the invention. Another aspect of the invention concerns pharmaceutical composition comprising the vectors or host cells of the invention, and a pharmaceutically acceptable carrier.
摘要翻译：本发明人涉及携带截短的嵌合CMV-鸡β-肌动蛋白（smCBA）启动子的载体，其中杂交的鸡β-肌动蛋白/兔β-珠蛋白内含子被大大缩短，并且它们用于递送至可操作连接的多核苷酸至宿主细胞在体外或体内，导致多核苷酸在宿主细胞中的表达。在一个实施方案中，携带smCBA启动子的载体被施用于眼睛。在另一个实施方案中，携带smCBA启动子的载体是自身互补的腺相关病毒（AAV）。 AAV载体可以是任何血清型（例如，1型，2型，3型，4型，5型，6型，7型，8型，9型，10型）。在另一个实施方案中，将携带smCBA启动子的自身互补载体施用于眼睛。本发明的另一方面涉及携带本发明载体的宿主细胞。本发明的另一方面涉及包含本发明的载体或宿主细胞的药物组合物和药学上可接受的载体。

78. 发明申请

WO2006116267A3 CELL-SPECIFIC MOLECULE AND METHOD FOR IMPORTING DNA INTO OSTEOBLAST NUCLEI 审中-公开
标题翻译：细胞特异性分子和将DNA导入到OSTEOBLAST NUCLEI中的方法
公开(公告)号：WO2006116267A3
公开(公告)日：2007-01-04
申请号：PCT/US2006015405
申请日：2006-04-24
申请人： GOVERNMENT OF THE US DEPT OF V , STRONG DONNA D , LINKHART THOMAS A , DEAN DAVID A
发明人： STRONG DONNA D , LINKHART THOMAS A , DEAN DAVID A
IPC分类号： C12N15/00
CPC分类号： A61K48/0058 , C07K14/4702 , C07K2319/09 , C12N15/85 , C12N2800/30 , C12N2830/001 , C12N2830/008 , C12N2830/15 , C12N2830/42 , C12N2830/85 , C12N2840/20
摘要： A plasmid, viral or linear DNA molecule containing a nucleic acid sequence derived from the promoter region of the hColla2 gene, which is selectively transported into the nuclei of cells in the osteoblast lineage. The sequence can be used independently as a nuclear entry sequence only, and/or as a nuclear entry sequence without regard to position, in a vector or linear DNA that directs gene expression and nuclear entry. The disclosure further includes a chimeric DNA sequence derived by the addition of osteoblast-specific enhancer sequences to the nuclear entry sequence/promoter sequence, to increase osteoblast-specific expression while retaining osteoblast-specific nuclear import. An enhancer sequence is derived from the promoter region of the human Core Binding Factor alpha 1 (Cbfal/Runx2) gene. The Cbfal/Runx2 promoter can be added to the sequence derived from, or alternatively, comprising the promoter region of the hColla2 gene. Also provided are methods of use of the novel sequences.
摘要翻译：含有衍生自hColla2基因的启动子区域的核酸序列的质粒，病毒或线性DNA分子，其被选择性转运到成骨细胞谱系中的细胞核中。该序列可以独立地用作核进入序列，和/或作为指导基因表达和核进入的载体或线性DNA中不考虑位置的核进入序列。本公开还包括通过向核进入序列/启动子序列添加成骨细胞特异性增强子序列而衍生的嵌合DNA序列，以增加成骨细胞特异性表达，同时保留成骨细胞特异性核进入。增强子序列衍生自人核心结合因子α1（Cbfal / Runx2）基因的启动子区。可以将Cbfal / Runx2启动子加入到由hColla2基因的启动子区域或另外包含hColla2基因的启动子区域的序列中。还提供了使用新序列的方法。

79. 发明申请

WO2006127980A2 OPTIMIZED CORE PROMOTERS AND USES THEREFOR 审中-公开
标题翻译：优化的核心促销者及其用途
公开(公告)号：WO2006127980A2
公开(公告)日：2006-11-30
申请号：PCT/US2006020394
申请日：2006-05-25
申请人： UNIV CALIFORNIA , KADONAGA JAMES T , GERSHON TAMAR
发明人： KADONAGA JAMES T , GERSHON TAMAR
IPC分类号： C07H21/04
CPC分类号： C07H21/04 , C12N15/85 , C12N2830/00 , C12N2830/15 , C12N2830/34
摘要： The present invention provides core promoter motif ten elements (MTE) and core promoter constructs comprising the MTEs and an initiator element (Inr) in combination with one or both of a TATA box and a downstream promoter element (DPE) which increases gene expression over the strongest known core promoters. Particularly, an optimized or super core promoter is provided which comprises Inr, MTE, TATA box and DPE elements. The present invention also provides expression vectors and host cells comprising the core promoter constructs. Additionally, methods of increasing production of a protein using the core promoter constructs are provided.
摘要翻译：本发明提供核心启动子基序十元素（MTE）和包含MTE的核心启动子构建体和与TATA盒和下游启动子元件（DPE）中的一个或两个组合的引发剂元件（Inr），其增加基因表达最强的核心推动者。特别地，提供了优化的或超核心启动子，其包括Inr，MTE，TATA盒和DPE元件。本发明还提供了包含核心启动子构建体的表达载体和宿主细胞。另外，提供了使用核心启动子构建体增加蛋白质生产的方法。

80. 发明申请

WO2006047250A3 USE OF APOPTOSIS INHIBITING COMPOUNDS IN DEGENERATIVE NEUROLOGICAL DISORDERS 审中-公开
标题翻译：使用抗病毒抑制化合物在衰老性神经疾病中的作用
公开(公告)号：WO2006047250A3
公开(公告)日：2006-11-23
申请号：PCT/US2005037822
申请日：2005-10-21
申请人： NEUROLOGIX INC , KAPLITT MICHAEL , MOUSSATOV SERGUEI
发明人： KAPLITT MICHAEL , MOUSSATOV SERGUEI
IPC分类号： A61P25/28 , A61K48/00 , C07K19/00 , C12N15/86
CPC分类号： C12N15/86 , A61K48/00 , C07K14/4747 , C07K2319/02 , C12N9/93 , C12N2750/14111 , C12N2750/14143 , C12N2800/107 , C12N2830/15 , C12N2830/48
摘要： The invention provides methods and compositions for localized delivery of a vector comprising a therapeutic agent to a specific region of the brain associated with a neurodegenerative diseases that is characterized by an excess buildup of intracellular protein aggregates. In particular, the invention provides methods and compositions used to deliver an adeno-associated virus vector (AAV) comprising a nucleotide sequence encoding an inhibitor of apoptosis protein (IAP) to cells in the region.
摘要翻译：本发明提供了用于将包含治疗剂的载体局部递送至与神经变性疾病相关的特定区域的方法和组合物，所述神经变性疾病的特征在于细胞内蛋白质聚集体的过量累积。特别地，本发明提供了用于将包含编码凋亡蛋白（IAP）抑制剂的核苷酸序列的腺相关病毒载体（AAV）递送至该区域的细胞的方法和组合物。

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