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51. 发明申请

US20040033601A1 Gene regulatory networks and methods of interdiction for controlling the differentiation state of a cell 审中-公开
标题翻译：用于控制细胞分化状态的基因调控网络和阻断方法
公开(公告)号：US20040033601A1
公开(公告)日：2004-02-19
申请号：US10452766
申请日：2003-05-30
发明人： Eric H. Davidson
IPC分类号： C12N015/85
CPC分类号： C12N15/63 , C12Q1/68
摘要： The invention provides a method of modulating a regulatory state of a cell. The method consists of: (a) identifying a point of interdiction within a cis regulatory network specifying a genetic regulatory architecture of a cell, and (b) introducing into a progenitor cell two or more network elements within said network to induce a predetermined series of cis regulatory network interactions resulting in a specified regulatory state of said progenitor cell. Also provided is a method of modulating a regulatory state of a cell. The method consists of: (a) identifying a point of interdiction within a cis regulatory network specifying a genetic regulatory architecture of a cellular state, and (b) introducing into a progenitor cell two or more network elements within said network to induce a predetermined series of cis regulatory network interactions resulting in a specified regulatory state of said progenitor cell. A cell having a specified regulatory state consisting of a modified genetic regulatory architecture is further provided. Methods of diagnosing and methods of treating an individual suffering from a cellular defect also are provided. The invention additionally provides a method of identifying a compound having differentiation or cell fate inducing activity.
摘要翻译：本发明提供调节细胞调节状态的方法。该方法包括：（a）在指定细胞的遗传调控结构的顺式调控网络内识别停顿点，以及（b）将所述网络内的两个或多个网络元件引入祖细胞，以诱导预定序列顺式调节网络相互作用导致所述祖细胞的特定调节状态。还提供了调节细胞调节状态的方法。该方法包括：（a）在规定细胞状态的遗传调控结构的顺式调控网络中识别停顿点，以及（b）将所述网络内的两个或多个网络元件引入到祖细胞中以诱导预定序列的顺式调节网络相互作用，导致所述祖细胞的特定调节状态。还提供具有由修饰的遗传调控结构组成的特定调节状态的细胞。还提供了诊断方法和治疗患有细胞缺陷的个体的方法。本发明另外提供了鉴定具有分化或细胞命运诱导活性的化合物的方法。

52. 发明申请

US20040033479A1 Method for generating competent or transformed cells 审中-公开
标题翻译：产生能量或转化细胞的方法
公开(公告)号：US20040033479A1
公开(公告)日：2004-02-19
申请号：US10451179
申请日：2003-06-20
发明人： Michael Rayner-Brandes , Ian David Watkins
IPC分类号： A01N001/02 , C12N015/85
CPC分类号： C12N15/87
摘要： This invention relates to rapid and simple procedures for preparing competent cells from dried non-competent prokaryotic cells. The dried non-competent cells can be stored for long periods at a wide range of temperatures prior to use in these procedures. The invention also relates to procedures for simultaneously or subsequently transforming said competent cells.
摘要翻译：本发明涉及从干燥的非感受态原核细胞制备感受态细胞的快速和简单的方法。干燥的无感受态细胞可以在这些程序使用之前在宽的温度范围内长时间储存。本发明还涉及同时或随后转化所述感受态细胞的方法。

53. 发明申请

US20040014217A1 Methods of transfection 审中-公开
标题翻译：转染方法
公开(公告)号：US20040014217A1
公开(公告)日：2004-01-22
申请号：US10296879
申请日：2003-06-02
发明人： Stephen Lewis Hart
IPC分类号： A61K048/00 , A61K039/395 , C12N015/85
CPC分类号： C12N15/87 , A61K2039/53
摘要： Transfection of confluent cells or other slowly dividing or non-dividing cells that are in contact with each other with a nucleic acid using a non-viral receptor targeted vector may be improved by the concurrent use of an agent that disrupts cell-cell junctions, especially EGTA. The vector is especially an integrin-targeting transfection vector complex comprising (i) a nucleic acid, especially a nucleic acid encoding a sequence of interest, (ii) an integrin-binding component, especially an integrin-targeting peptide, (iii) a polycationic nucleic acid-binding component, especially an oligolysine, and (iv) a lipid component, especially, DOPE, DOTMA, DOSPA or combinations thereof. Various applications of the improved method of transfection are described.
摘要翻译：特别是通过使用非病毒受体靶向载体与核酸相互接触的汇合细胞或其他缓慢分裂或非分裂细胞的转染，可以通过同时使用破坏细胞 - 细胞连接的试剂 EGTA。载体特别是整合素靶向转染载体复合物，其包含（i）核酸，特别是编码感兴趣序列的核酸，（ii）整联蛋白结合组分，特别是整联蛋白靶向肽，（iii）聚阳离子核酸结合组分，特别是寡聚赖氨酸，和（iv）脂质组分，特别是DOPE，DOTMA，DOSPA或其组合。描述了改进的转染方法的各种应用。

54. 发明申请

US20040014025A1 Method of effecting a change in a cell, and a vector 有权
标题翻译：影响细胞变化的方法和载体
公开(公告)号：US20040014025A1
公开(公告)日：2004-01-22
申请号：US10300666
申请日：2002-11-19
发明人： Paul Jan J. Hooykaas , Annette Caroline Vergunst , Barbara Schrammeijer
IPC分类号： C12Q001/00 , C12N015/85
CPC分类号： C12N15/62 , C07K14/195 , C07K2319/00 , C12N9/00 , C12N15/8205
摘要： A method of effecting a change in a cell using a transfer system which is contacted with the cell to be changed. The transfer system comprises a fusion protein which is transferred into the cell using the VirB/VirD4 transfer system. According to the invention a fusion protein BA is introduced into the cell to be changed comprising i) as a first part A an oligopeptide comprising the C-terminal amino acids 1-20 of VirF, VirD2, VirE2, VirE3, VirD5 or MobA, or an analogue thereof and ii) as a second part B a polypeptide capable of effecting a cell-changing activity in the cell to be changed, wherein the polypeptide is attached with the C-terminal end thereof with the N-terminal end of the first part.
摘要翻译：使用与要改变的单元接触的传送系统来实现单元的改变的方法。转移系统包含融合蛋白，其使用VirB / VirD4转移系统转移到细胞中。根据本发明，将融合蛋白BA引入要改变的细胞中，包括i）作为第一部分A的包含VirF，VirD2，VirE2，VirE3，VirD5或MobA的C末端氨基酸1-20的寡肽或其类似物和ii）作为第二部分B，能够在待改变的细胞中实现细胞变化活性的多肽，其中所述多肽与其C末端连接有第一部分的N末端。

55. 发明申请

US20040009934A1 GM-CSF nucleic acid sequences 失效
公开(公告)号：US20040009934A1
公开(公告)日：2004-01-15
申请号：US10188056
申请日：2002-07-03
发明人： Jian-Tai Qiu , Wan-Ching Lai , Yong Liang Chu , Frank Q. Li
IPC分类号： A61K048/00 , C07H021/04 , C12N015/85 , C12P021/02
CPC分类号： C07K14/535 , A61K2039/55522 , C07H21/04
摘要： The present invention relates to improved methods for the treatment or prevention of disease with gene therapy or vaccines comprising host optimized GM-CSF nucleic acid sequences. More particularly, the invention provides methods for codon optimized GM-CSF with enhanced expression and adjuvant activity ex vivo and in vivo. The present invention also provides for methods of optimizing homologous gene expression ex vivo and in vitro or in transgenic animals.

56. 发明申请

US20040009898A1 Targeting vector to the urokinase plasminogen activator receptor 有权
标题翻译：靶向载体到尿激酶纤溶酶原激活物受体
公开(公告)号：US20040009898A1
公开(公告)日：2004-01-15
申请号：US10442880
申请日：2003-05-21
发明人： Michael J. Welsh , Paola T. Drapkin
IPC分类号： A61K048/00 , A61K038/28 , C12N015/85
CPC分类号： C12N9/6462 , A61K31/70 , A61K38/49 , C07K2319/00 , C12Y304/21073
摘要： The present invention relates to the targeted delivery of a delivery vehicle construct which specifically binds to and stimulates endocytosis into cells expressing the urokinase plasminogen activator receptor (uPAR), and particularly human airway epithelia. The delivery vehicle construct comprises a portion of uPA and a cargo linked thereto and is useful for the targeted delivery of the cargo to a cell. In one aspect of the invention, the uPA portion of the delivery vehicle construct comprises the wild-type uPA, a fragment of uPA which has the PAI-1 binding region deleted, or a uPA peptide comprising amino acids 13-19 and is useful for the targeted delivery of the cargo to cells, and in particular to airway epithelia. The present invention also provides a method for delivering the delivery vehicle construct to a cell. The method comprises the steps of (a) contacting a target cell with a delivery vehicle construct comprising a uPA portion and a cargo portion; and (b) obtaining a desired result in the target cell.
摘要翻译：本发明涉及特异性结合并刺激表达尿激酶纤溶酶原激活物受体（uPAR）的细胞，特别是人气道上皮的细胞的递送载体构建体的靶向递送。输送车辆构造包括uPA的一部分和与其相关联的货物，并且可用于将货物有目标地运送到电池。在本发明的一个方面，递送载体构建体的uPA部分包含野生型uPA，具有缺失的PAI-1结合区的uPA的片段或包含氨基酸13-19的uPA肽，并且可用于将货物有目标地运送到细胞，特别是气道上皮细胞。本发明还提供了一种用于将递送载体构建体递送到细胞的方法。该方法包括以下步骤：（a）使目标细胞与包含uPA部分和货物部分的递送载体构建体接触; 和（b）在靶细胞中获得期望的结果。

57. 发明申请

US20030237102A1 Method of reducing neuronal electrical activity with a potassium channel subunit 失效
标题翻译：用钾通道亚基减少神经元电活动的方法
公开(公告)号：US20030237102A1
公开(公告)日：2003-12-25
申请号：US10438753
申请日：2003-05-15
发明人： Michael N. Nitabach , Justin Blau , Todd C. Holmes , Steven A. N. Goldstein
IPC分类号： A01K067/00 , A01K067/033 , A01K067/027 , C07H021/04 , C12N015/85 , C12N005/06
CPC分类号： C12N15/8509 , A01K67/0333 , A01K67/0339 , A01K2217/05 , A01K2227/102 , A01K2227/103 , A01K2227/105 , A01K2227/106 , A01K2227/107 , A01K2227/108 , A01K2267/0306 , A61K48/00 , C07K14/705
摘要： Methods of reducing the excitability of an excitable cell by transforming an excitable cell with a nucleic acid construct encoding an open rectifier Knull channel (dORK) (SEQ ID NO:2) or a modified open rectifier Knull channel (dORKnull) (SEQ ID NO:4), and expressing the open rectifier Knull channel in the excitable cell, wherein the excitability of the transformed cell is reduced. Also featured are transgenic animals expressing dORK or dORKnull.
摘要翻译：通过用编码开放整流体K +通道（dORK）（SEQ ID NO：2）或修饰的开放式整流器K +通道（dORKDelta）的核酸构建体转化可兴奋细胞来降低兴奋性细胞的兴奋性的方法）（SEQ ID NO：4），并且在可兴奋细胞中表达开放整流体K +通道，其中转化细胞的兴奋性降低。还特征是表达dORK或dORKDelta的转基因动物。

58. 发明申请

US20030236208A1 Targeted chromosomal genomic alterations in plants using modified single stranded oligonucleotides 审中-公开
标题翻译：使用修饰的单链寡核苷酸的植物中靶向的染色体基因组改变
公开(公告)号：US20030236208A1
公开(公告)日：2003-12-25
申请号：US10307005
申请日：2002-11-26
发明人： Eric B. Kmiec , Howard B. Gamper , Michael C. Rice , Jungsup Kim
IPC分类号： A61K048/00 , C07H021/04 , C12N015/85
CPC分类号： C12N15/113 , A61K38/00 , A61K48/00 , C12N15/102 , C12N15/8213 , C12N2310/315 , C12N2310/321 , C12N2310/3231 , C12N2310/346 , Y02A50/473 , C12N2310/3521
摘要： Presented are methods and compositions for targeted chromosomal genomic alterations with modified single-stranded oligonucleotides. The oligonucleotides of the invention have modified nuclease-resistant termini comprising LNA, phosphorothioate linkages or 2null-O-Me base analogues or combinations of such modifications.
摘要翻译：提出了用修饰的单链寡核苷酸靶向染色体基因组改变的方法和组合物。本发明的寡核苷酸具有包含LNA，硫代磷酸酯键或2'-O-Me碱基类似物或这些修饰的组合的修饰的核酸酶抗性末端。

59. 发明申请

US20030229034A1 Multi-component biological transport systems 有权
标题翻译：多组分生物运输系统
公开(公告)号：US20030229034A1
公开(公告)日：2003-12-11
申请号：US09910432
申请日：2001-07-20
申请人： Essentia Biosystems, Inc.
发明人： Jacob Waugh , Michael Dake
IPC分类号： A61K048/00 , A61K039/08 , C12N015/85
CPC分类号： B82Y5/00 , A61K47/645 , A61K47/665 , A61K49/128
摘要： Compositions and methods are provided that are useful for the delivery of therapeutic agents, including nucleic acids. The compositions can be prepared with components useful for targeting the delivery of the compositions as well as imaging components.
摘要翻译：提供了可用于递送包括核酸在内的治疗剂的组合物和方法。组合物可以用可用于靶向组合物递送以及成像组分的组分制备。

60. 发明申请

US20030228694A1 Transfection method and uses related thereto 有权
标题翻译：转染方法及其用途
公开(公告)号：US20030228694A1
公开(公告)日：2003-12-11
申请号：US10379130
申请日：2003-03-04
发明人： David M. Sabatini
IPC分类号： C12N015/85 , C12N015/86
CPC分类号： C12Q1/6806 , C12N15/87 , C12N15/88 , C12Q2565/518
摘要： The invention features a method of introducing nucleic acid molecules into eukaryotic cells by (a) depositing a nucleic acid molecule-containing mixture onto a surface, (b) affixing the nucleic acid molecule-containing mixture to the surface, and (c) plating eukaryotic cells onto the surface under appropriate conditions for entry of the nucleic acid molecules into the cells.
摘要翻译：本发明的特征在于通过以下步骤将核酸分子引入真核细胞：（a）将含核酸分子的混合物沉积在表面上，（b）将含核酸分子的混合物固定到表面，和（c）真核细胞细胞在适当条件下进入表面，以使核酸分子进入细胞。

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