公开(公告)号：US08470528B2

公开(公告)日：2013-06-25

申请号：US12771390

申请日：2010-04-30

申请人： Renata Pasqualini ,  Wadih Arap ,  Juri Gelovani ,  Frank C. Marini, III ,  Amin Hajitou ,  Mian Alauddin ,  Martin Trepel

发明人： Renata Pasqualini ,  Wadih Arap ,  Juri Gelovani ,  Frank C. Marini, III ,  Amin Hajitou ,  Mian Alauddin ,  Martin Trepel

IPC分类号： C12Q1/70 ,  G01N33/53 ,  A61K51/00 ,  A61K49/00 ,  A61B5/055

CPC分类号： A61K47/6455 ,  A61K47/62 ,  A61K47/6901 ,  A61K48/00 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2795/14143 ,  C12N2810/405 ,  C12N2810/85

摘要： Embodiments of the invention are generally directed to compositions and methods of delivering one or more transgene to a target cell, such as a tumor cell, in a site-specific manner to achieve enhanced expression and to constructs and compositions useful in such applications. In certain aspects, expression from a therapeutic nucleic acid may be assessed prior to administration of a treatment or diagnostic procedure to or on a subject.

摘要翻译： 本发明的实施方案通常涉及以一种位点特异性方式将一种或多种转基因递送至靶细胞例如肿瘤细胞以实现增强的表达的组合物和方法以及在这些应用中有用的构建体和组合物。 在某些方面，可以在向受试者施用治疗或诊断程序之前评估来自治疗性核酸的表达。

公开(公告)号：US20120178903A1

公开(公告)日：2012-07-12

申请号：US13403765

申请日：2012-02-23

申请人： Renata Pasqualini ,  Wadih Arap ,  Juri Gelovani ,  Amin Hajitou ,  Mian Alauddin ,  Martin Trepel

发明人： Renata Pasqualini ,  Wadih Arap ,  Juri Gelovani ,  Amin Hajitou ,  Mian Alauddin ,  Martin Trepel

IPC分类号： C07K7/06

CPC分类号： A61K47/6455 ,  A61K47/62 ,  A61K47/6901 ,  A61K48/00 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2795/14143 ,  C12N2810/405 ,  C12N2810/85

摘要： Embodiments of the invention are generally directed to compositions and methods of delivering one or more transgene to a target cell, such as a tumor cell, in a site-specific manner to achieve enhanced expression and to constructs and compositions useful in such applications. In certain aspects, expression from a therapeutic nucleic acid may be assessed prior to administration of a treatment or diagnostic procedure to or on a subject.

摘要翻译： 本发明的实施方案通常涉及以一种位点特异性方式将一种或多种转基因递送至靶细胞例如肿瘤细胞以实现增强的表达的组合物和方法以及在这些应用中有用的构建体和组合物。 在某些方面，可以在向受试者施用治疗或诊断程序之前评估来自治疗性核酸的表达。

公开(公告)号：US20100016413A1

公开(公告)日：2010-01-21

申请号：US12446976

申请日：2007-11-05

申请人： Frederick L. Hall ,  John P. Levy ,  Rebecca A. Reed ,  Erlinda M. Gordon

发明人： Frederick L. Hall ,  John P. Levy ,  Rebecca A. Reed ,  Erlinda M. Gordon

IPC分类号： A61K31/7088 ,  C12N15/63 ,  A61P35/00

CPC分类号： A61K31/7088 ,  A61K35/76 ,  A61K38/1709 ,  A61K38/193 ,  A61K38/45 ,  A61K48/00 ,  C12N15/86 ,  C12N2740/13043 ,  C12N2740/13045 ,  C12N2810/85 ,  Y02A50/473 ,  A61K2300/00

摘要： Systems for pathotropic (disease-seeking) targeted gene delivery are provided, including viral particles with extremely high titers. In particular, the viral particles are engineered to specifically deliver therapeutic or diagnostic agents to a disease site, such as cancer metastic sites. Personalized dosing regimens are also provided to treat diseases such as cancer efficaciously with reduced adverse side effects.

摘要翻译： 提供用于致病性（寻疾）靶向基因递送的系统，包括具有极高滴度的病毒颗粒。 特别地，病毒颗粒被工程化以将治疗或诊断剂特异性递送到疾病部位，例如癌症转移部位。 还提供个性化的给药方案以有效地治疗诸如癌症的疾病，减少不良副作用。

公开(公告)号：US20100003197A1

公开(公告)日：2010-01-07

申请号：US12378100

申请日：2009-02-11

申请人： Malavosklish Bikram

发明人： Malavosklish Bikram

IPC分类号： A61K49/06 ,  A61P35/00

CPC分类号： A61K33/24 ,  A61K33/26 ,  A61K33/32 ,  A61K41/0052 ,  A61K47/6923 ,  A61K49/183 ,  A61K49/1848 ,  A61K49/186 ,  A61K49/1866 ,  B82Y5/00 ,  C12N15/87 ,  C12N2740/16322 ,  C12N2810/6054 ,  C12N2810/85 ,  Y10T428/2982 ,  Y10T428/2991

摘要： The present invention provides biomimetic contrast agents, dual functional contrast agents effective for therapeutic gene delivery and magnetic nanoparticles which comprise functionalized iron oxide nanoparticle cores, one of an inert gold layer, a layer of inert metal seeds or a silica layer and, optionally, one or both of an outer gold-silver nanoshell or a targeting ligand attached to the inert gold layer or the gold-silver nanoshell. Also provided are methods of in vivo magnetic resonance imaging, of treating primary or metastatic cancers or of ablating atherosclerotic plaque using the contrast agents and magnetic particles. In addition, kits comprising the biomimetic contrast agents, dual contrast agents and magnetic nanoparticles.

摘要翻译： 本发明提供仿生造影剂，对治疗性基因递送有效的双重功能造影剂和包含官能化的氧化铁纳米颗粒芯，惰性金层，惰性金属晶种层或二氧化硅层之一的磁性纳米颗粒，以及任选的一种 或外金银纳米壳或连接到惰性金层或金 - 银纳米壳的靶向配体两者。 还提供了体内磁共振成像，使用造影剂和磁性颗粒治疗原发性或转移性癌症或消融动脉粥样硬化斑块的方法。 另外，包含仿生造影剂，双重造影剂和磁性纳米颗粒的试剂盒。

公开(公告)号：US20090191261A1

公开(公告)日：2009-07-30

申请号：US12292683

申请日：2008-11-24

申请人： Liang Xu ,  Cheng-Cheng Huang ,  William Alexander ,  WenHua Tang ,  Esther H. Chang

发明人： Liang Xu ,  Cheng-Cheng Huang ,  William Alexander ,  WenHua Tang ,  Esther H. Chang

IPC分类号： A61K9/127 ,  A61K39/395

CPC分类号： C12N15/86 ,  A61K9/0019 ,  A61K9/1271 ,  A61K9/1272 ,  A61K47/6849 ,  A61K47/6913 ,  A61K48/0008 ,  C12N2710/10343 ,  C12N2810/85 ,  C12N2810/859

摘要： Nucleic acid-immunoliposome compositions useful as therapeutic agents are disclosed. These compositions preferably comprise (i) cationic liposomes, (ii) a single chain antibody fragment which binds to a transferrin receptor, and (iii) a nucleic acid encoding a wild type p53. These compositions target cells which express transferrin receptors, e.g., cancer cells. These compositions can be used therapeutically to treat persons or animals who have cancer, e.g., head and neck cancer, breast cancer or prostate cancer.

公开(公告)号：US20080112929A1

公开(公告)日：2008-05-15

申请号：US11947771

申请日：2007-11-30

申请人： Imre Kovesdi ,  Susan Hedley ,  Nikolay Korokhov

发明人： Imre Kovesdi ,  Susan Hedley ,  Nikolay Korokhov

IPC分类号： A61K48/00 ,  C12N15/861 ,  C07K14/075

CPC分类号： C12N15/86 ,  A61K48/00 ,  C07K2319/30 ,  C07K2319/31 ,  C07K2319/33 ,  C07K2319/74 ,  C12N7/00 ,  C12N2710/10343 ,  C12N2710/10345 ,  C12N2810/85 ,  C12N2810/859 ,  C12N2830/008

摘要： The present invention encompasses replication deficient or a replication competent adenoviral vectors which may comprise moieties covering and shielding the vector from the effects of humoral immune responses, as well as a method of constructing and using such vectors. The preferred viral constructs may incorporate the shielding moieties into the pIX coat protein of the adenovirus vectors. The invention also provides recombinant viral vectors with both shielding and specific targeting abilities. Preferably, the viral vector may comprise a nucleic acid sequence, which codes for therapeutically important genes. Methods for treating of a host with an effective amount of adenovirus vector of the present invention are also provided.

摘要翻译： 本发明包括复制缺陷型或复制型腺病毒载体，其可以包含覆盖和屏蔽载体免受体液免疫应答影响的部分，以及构建和使用这些载体的方法。 优选的病毒构建体可以将屏蔽部分并入腺病毒载体的pIX外壳蛋白中。 本发明还提供具有屏蔽和特异性靶向能力的重组病毒载体。 优选地，病毒载体可以包含编码治疗重要基因的核酸序列。 还提供了用有效量的本发明的腺病毒载体处理宿主的方法。

公开(公告)号：US20080071068A1

公开(公告)日：2008-03-20

申请号：US10589955

申请日：2005-02-21

申请人： Hideki Oba ,  Masayuki Fujii

发明人： Hideki Oba ,  Masayuki Fujii

IPC分类号： C07H21/02 ,  C07H21/04

CPC分类号： C07K14/005 ,  A61K47/545 ,  A61K47/62 ,  C07K2319/09 ,  C07K2319/095 ,  C07K2319/10 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/351 ,  C12N2310/3513 ,  C12N2320/32 ,  C12N2740/16122 ,  C12N2740/16322 ,  C12N2810/6009 ,  C12N2810/6054 ,  C12N2810/85

摘要： It is intended to provide a modified DNA or RNA, the cytoplasmic localization of which has been established, and an siRNA, which is localized in the cytoplasm, shows a high activity and, therefore, is appropriately usable as a genetic drug, by using a means generally applicable to DNAs of various types regardless of original tissues. A cytoplasmic localization DNA or RNA modified with a peptide can be constructed by modifying a DNA fragment with an active hydrogen-containing group on a solid support, fusing a peptide having an active hydrogen-containing group therewith and then removing from the solid support. On the other hand, a cytoplasmic localization siRNA can be obtained by introducing chemical modification group(s) into the 5′-terminus of at least one of the sense chain and the antisense chain constituting the double-strand, or a dangling end of the antisense chain, or both of them.

摘要翻译： 旨在提供其已经建立的细胞质定位的修饰的DNA或RNA，并且位于细胞质中的siRNA显示高活性，因此可以适当地用作遗传药物，通过使用 通常适用于各种类型的DNA，而不管原始组织。 用肽修饰的细胞质定位DNA或RNA可以通过在固体支持物上用含活性氢基团修饰DNA片段来构建，将具有活性含氢基团的肽与其固定，然后从固体支持物上除去。 另一方面，可以通过将化学修饰基团引入到构成双链的有义链和反义链中的至少一个的5'末端或者悬挂的两端的悬挂末端来获得细胞质定位siRNA 反义链，或它们两者。

公开(公告)号：US20080019988A1

公开(公告)日：2008-01-24

申请号：US11713514

申请日：2007-03-01

申请人： Juha Punnonen ,  Willem Stemmer ,  Russell Howard ,  Phillip Patten

发明人： Juha Punnonen ,  Willem Stemmer ,  Russell Howard ,  Phillip Patten

IPC分类号： A61K39/12 ,  A61P31/12 ,  C07K14/00 ,  C12N15/09

CPC分类号： A61K39/385 ,  A61K39/00 ,  A61K2039/53 ,  A61K2039/6037 ,  A61K2039/605 ,  C12N15/1037 ,  C12N15/85 ,  C12N2810/50 ,  C12N2810/85 ,  C12N2810/859

摘要： This invention provides methods of obtaining reagents for increasing the specificity of genetic vaccines for a desired target cell or tissue type. The invention also provides delivery vehicles for use to improve genetic vaccine specificity for a target cell or tissue type.

摘要翻译： 本发明提供获得用于增加所需靶细胞或组织类型的遗传疫苗的特异性的试剂的方法。 本发明还提供用于改善靶细胞或组织类型的遗传疫苗特异性的递送载体。

公开(公告)号：US07026462B2

公开(公告)日：2006-04-11

申请号：US10006069

申请日：2001-12-06

申请人： Edward Rebar ,  Andrew Jamieson ,  Qiang Liu ,  Pei-Qi Liu ,  Elizabeth J. Wolffe ,  Stephen P. Eisenberg ,  Eric Jarvis

发明人： Alan Wolffe

IPC分类号： C07H21/02 ,  A01N43/04

CPC分类号： A61K48/005 ,  A61K38/00 ,  A61K48/00 ,  C07K14/47 ,  C07K14/4702 ,  C07K14/52 ,  C07K2319/00 ,  C12N15/86 ,  C12N2710/10343 ,  C12N2740/13043 ,  C12N2799/021 ,  C12N2810/85 ,  C12N2830/008

摘要： Provided herein are a variety of methods and compositions for regulating angiogenesis, such methods and compositions being useful in a variety of applications where modulation of vascular formation is useful, including, but not limited to, treatments for ischemia and wound healing. Certain of the methods and compositions accomplish this by using various zinc finger proteins that bind to particular target sites in one or more VEGF genes. Nucleic acids encoding the zinc finger proteins are also disclosed. Methods for modulating the expression of one or more VEGF genes with the zinc finger proteins and nucleic acids are also disclosed. Such methods can also be utilized in a variety of therapeutic applications that involve the regulation of endothelial cell growth. Pharmaceutical compositions including the zinc finger proteins or nucleic acids encoding them are also provided.

公开(公告)号：US20050267062A1

公开(公告)日：2005-12-01

申请号：US11115922

申请日：2005-04-26

申请人： Edward Rebar ,  Andrew Jamieson ,  Qiang Liu ,  Pei-Qi Liu ,  Alan Wolffe ,  Stephen Eisenberg ,  Eric Jarvis

发明人： Edward Rebar ,  Andrew Jamieson ,  Qiang Liu ,  Pei-Qi Liu ,  Alan Wolffe ,  Stephen Eisenberg ,  Eric Jarvis

IPC分类号： C12N15/09 ,  A61K38/00 ,  A61K48/00 ,  A61P9/00 ,  A61P9/10 ,  A61P17/02 ,  A61P19/02 ,  A61P29/00 ,  A61P35/00 ,  A61P43/00 ,  C07K14/47 ,  C07K14/52 ,  C12N15/12 ,  C12N15/861 ,  C12N15/867 ,  C12Q1/02

CPC分类号： A61K48/005 ,  A61K38/00 ,  A61K48/00 ,  C07K14/47 ,  C07K14/4702 ,  C07K14/52 ,  C07K2319/00 ,  C12N15/86 ,  C12N2710/10343 ,  C12N2740/13043 ,  C12N2799/021 ,  C12N2810/85 ,  C12N2830/008

摘要： Provided herein are a variety of methods and compositions for regulating angiogenesis, such methods and compositions being useful in a variety of applications where modulation of vascular formation is useful, including, but not limited to, treatments for ischemia and wound healing. Certain of the methods and compositions accomplish this by using various zinc finger proteins that bind to particular target sites in one or more VEGF genes. Nucleic acids encoding the zinc finger proteins are also disclosed. Methods for modulating the expression of one or more VEGF genes with the zinc finger proteins and nucleic acids are also disclosed. Such methods can also be utilized in a variety of therapeutic applications that involve the regulation of endothelial cell growth. Pharmaceutical compositions including the zinc finger proteins or nucleic acids encoding them are also provided.

摘要翻译： 本文提供了用于调节血管生成的各种方法和组合物，所述方法和组合物可用于血管形成调节是有用的多种应用，包括但不限于缺血和伤口愈合的治疗。 某些方法和组合物通过使用结合一个或多个VEGF基因中的特定靶位点的各种锌指蛋白来实现。 还公开了编码锌指蛋白的核酸。 还公开了用锌指蛋白和核酸调节一种或多种VEGF基因的表达的方法。 这些方法也可用于涉及内皮细胞生长调节的各种治疗应用中。 还提供了包括锌指蛋白或编码它们的核酸的药物组合物。