公开(公告)号：WO2013025461A1

公开(公告)日：2013-02-21

申请号：PCT/US2012/050249

申请日：2012-08-10

Applicant: VIRXSYS CORPORATION ,  MCGARRITY, Gerard John

Inventor： MCGARRITY, Gerard John

IPC: C12N5/00 ,  C12N5/02 ,  A61K48/00 ,  C07H21/04

CPC classification number: C12N15/85 ,  A61K48/0066 ,  C07K14/4747 ,  C07K14/82 ,  C07K2319/31 ,  C12N15/111 ,  C12N15/86 ,  C12N2320/33 ,  C12N2740/16043 ,  C12N2750/14143 ,  C12N2800/108 ,  C12N2830/008 ,  C12N2840/445

Abstract: Methods and compositions are provided for generating novel nucleic acid molecules through targeted spliceosome mediated RNA trans -splicing (SMaRT TM ) that result in expression of a splicing isoform or variant thereof. The methods and compositions are based upon pre-trans-splicing molecules (PTMs) designed to interact with a target pre-mRNA molecule and mediate a t rans -splicing reaction generating a novel chimeric RNA molecule encoding a splicing isoform for the treatment of a variety of gene isoform induced diseases such as cancer.

Abstract translation: 提供了通过靶向剪接体介导的RNA转录（SMaRTTM）产生新的核酸分子的方法和组合物，其导致剪接同种型或其变体的表达。 所述方法和组合物基于设计为与靶前mRNA分子相互作用的转录前分子（PTM），并介导反剪接反应，产生编码剪接同种型的新型嵌合RNA分子，用于治疗各种 基因异构体诱导疾病如癌症。

公开(公告)号：WO2012066171A1

公开(公告)日：2012-05-24

申请号：PCT/ES2011/070780

申请日：2011-11-14

Applicant: UNIVERSIDADE DE SANTIAGO DE COMPOSTELA ,  UNIVERSIDAD DE VALLADOLID ,  PÁRRAGA MENESES, Jenny ,  KONAT ZORZI, Giovanni ,  PAOLICELLI, Patrizia ,  SEIJO REY, Begoña ,  SÁNCHEZ BARREIRO, Alejandro ,  CONTRERAS RUIZ, Laura ,  DIEBOLD LUQUE, Yolanda

Inventor： PÁRRAGA MENESES, Jenny ,  KONAT ZORZI, Giovanni ,  PAOLICELLI, Patrizia ,  SEIJO REY, Begoña ,  SÁNCHEZ BARREIRO, Alejandro ,  CONTRERAS RUIZ, Laura ,  DIEBOLD LUQUE, Yolanda

IPC: A61K9/51 ,  A61K48/00 ,  A61P27/02 ,  A61K47/36 ,  A61K47/22

CPC classification number: A61K9/0048 ,  A61K48/005 ,  A61K48/0091 ,  C12N15/85 ,  C12N15/88 ,  C12N2800/108 ,  C12N2820/60 ,  C12N2840/203

Abstract: La presente invención se refiere a nanopartículas poliméricas las cuales que comprenden un polinucleótido que codifica para una proteína MUC5AC modificada de modo que presenta el extremo N-terminal de MUC5AC unido al dominio de dimerización situado en el extremo C-terminal, o un plásmido o una construcción genética que lo contenga, y a su uso para la elaboración de un medicamento para la prevención y/o el tratamiento de una enfermedad que cursa con una deficiencia de mucinas. También se refiere a una composición farmacéutica que comprenda dichas nanopartículas y a su uso para la elaboración de un medicamento para la prevención y/o el tratamiento de una enfermedad que cursa con una deficiencia de mucinas.

Abstract translation: 本发明涉及聚合物纳米粒子，其包括编码用于修饰的MUC5AC蛋白的多核苷酸，使得MUC5AC的N末端连接到位于C末端的二聚化结构域，或质粒或含有 并且用于制备用于预防和/或治疗涉及粘蛋白缺乏症的药物。 它还涉及包含所述纳米颗粒的药物组合物及其用于制备用于预防和/或治疗涉及粘蛋白缺乏症的药物的用途。

公开(公告)号：WO2011071476A3

公开(公告)日：2011-12-08

申请号：PCT/US2009064488

申请日：2009-11-13

Applicant: LIFE TECHNOLOGIES CORP ,  LAKSHMIPATHY UMA ,  THYAGARAJAN BHASKAR ,  CHESNUT JONATHAN ,  ANEST VASILIKI ,  BENNETT ROBERT ,  LIEU PAULINE ,  HANSON GEORGE ,  THOMPSON DAVID ,  CHASE LUCAS ,  SHIPLEY GARY ,  WILSON ELIZABETH

Inventor： LAKSHMIPATHY UMA ,  THYAGARAJAN BHASKAR ,  CHESNUT JONATHAN ,  ANEST VASILIKI ,  BENNETT ROBERT ,  LIEU PAULINE ,  HANSON GEORGE ,  THOMPSON DAVID ,  CHASE LUCAS ,  SHIPLEY GARY ,  WILSON ELIZABETH

IPC: C12N15/86

CPC classification number: C12N15/86 ,  C12N2710/16221 ,  C12N2710/16222 ,  C12N2710/16243 ,  C12N2799/022 ,  C12N2799/026 ,  C12N2799/027 ,  C12N2800/108 ,  C12N2820/002 ,  C12N2820/007 ,  C12N2820/60

Abstract: The disclosure relates generally to genetic manipulation of stem and primary cells and to reprogramming of somatic cells, more specifically, human cells. In particular, compositions and methods are disclosed for the generation and maintenance of such engineered cells.

Abstract translation: 本公开内容通常涉及干细胞和原代细胞的基因操作以及体细胞，更具体而言是人细胞的重编程。 具体而言，公开了用于产生和维持这种工程细胞的组合物和方法。

公开(公告)号：WO2011094738A1

公开(公告)日：2011-08-04

申请号：PCT/US2011/023330

申请日：2011-02-01

Applicant: THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY ,  WU, Joseph ,  LONGAKER, Michael T. ,  KAY, Mark A. ,  SUNG, Ning ,  JIA, FangJun ,  CHEN, Zhi-Ying ,  PANETTA, Nicholas ,  GUPTA, Deepak

Inventor： WU, Joseph ,  LONGAKER, Michael T. ,  KAY, Mark A. ,  SUNG, Ning ,  JIA, FangJun ,  CHEN, Zhi-Ying ,  PANETTA, Nicholas ,  GUPTA, Deepak

IPC: C12N15/00 ,  C12N5/00

CPC classification number: C12N5/0696 ,  C12N15/85 ,  C12N2501/602 ,  C12N2501/603 ,  C12N2501/604 ,  C12N2501/605 ,  C12N2501/606 ,  C12N2506/1384 ,  C12N2510/00 ,  C12N2800/00 ,  C12N2800/108 ,  C12N2800/24

Abstract: Human somatic cells are reprogrammed to become induced pluripotent stem cells (iPS cells) by the introduction of a minicircle DNA vector. Cells of interest include adipose stem cells.

Abstract translation: 通过引入小环DNA载体，将人体细胞重新编程成诱导多能干细胞（iPS细胞）。 感兴趣的细胞包括脂肪干细胞。

公开(公告)号：WO2009152529A2

公开(公告)日：2009-12-17

申请号：PCT/US2009047423

申请日：2009-06-15

Applicant: WHITEHEAD BIOMEDICAL INST ,  JAENISCH RUDOLPH

Inventor： JAENISCH RUDOLPH

IPC: C12N15/12 ,  C12N5/10 ,  C12N15/867 ,  C12Q1/02

CPC classification number: C12N15/86 ,  A01K67/0271 ,  C07K14/4705 ,  C12N5/0606 ,  C12N5/0696 ,  C12N15/79 ,  C12N15/85 ,  C12N2501/60 ,  C12N2501/602 ,  C12N2501/603 ,  C12N2501/604 ,  C12N2501/605 ,  C12N2501/606 ,  C12N2501/608 ,  C12N2506/11 ,  C12N2506/115 ,  C12N2510/00 ,  C12N2740/15041 ,  C12N2740/15043 ,  C12N2740/16043 ,  C12N2799/027 ,  C12N2800/108 ,  C12N2840/203 ,  G01N33/5008

Abstract: The disclosure relates to a method of reprogrammlng one or more somatic cells, e.g., partially differentiated or fully/terminally differentiated somatic cells, to a less differentiated state, e.g., a pluripotent or multipotent state. In further embodiments the invention also relates to reprogrammed somatic cells produced by methods of the invention, to chimeric animals comprising reprogrammed somatic cells of the invention, to uses of said cells, and to methods for identifying agents useful for reprogramming somatic cells.

Abstract translation: 本公开涉及将一种或多种体细胞（例如部分分化或完全/终末分化的体细胞）重新编程为较少分化状态（例如多能或多能状态）的方法。 在进一步的实施方案中，本发明还涉及通过本发明的方法产生的重编程体细胞细胞，包含本发明的重编程体细胞的嵌合动物，所述细胞的用途以及用于鉴定用于重编程体细胞的药物的方法。

公开(公告)号：WO2009149233A1

公开(公告)日：2009-12-10

申请号：PCT/US2009/046209

申请日：2009-06-04

Applicant: STEM CELL PRODUCTS, INC. ,  MACK, Amanda ,  THOMSON, James

Inventor： MACK, Amanda ,  THOMSON, James

IPC: C12N5/08 ,  C12N5/10 ,  C12N15/869

CPC classification number: C12N5/0696 ,  C07K14/005 ,  C12N15/85 ,  C12N2501/602 ,  C12N2501/603 ,  C12N2501/604 ,  C12N2501/605 ,  C12N2501/606 ,  C12N2501/608 ,  C12N2510/00 ,  C12N2710/16222 ,  C12N2800/107 ,  C12N2800/108 ,  C12N2800/24 ,  C12N2820/60 ,  C12N2840/105 ,  C12N2840/206

Abstract: Methods and composition of induction of pluripotent stem cells and other desired cell types are disclosed. For example, in certain aspects methods for generating essentially vector-free induced pluripotent stem cells are described. Furthermore, the invention provides induced pluripotent stem cells and desired cell types essentially free of exogenous vector elements with the episomal expression vectors to express differentiation programming factors.

Abstract translation: 公开了诱导多能干细胞和其它所需细胞类型的方法和组成。 例如，在某些方面，描述了用于产生基本上无载体的诱导多能干细胞的方法。 此外，本发明提供诱导多能干细胞和基本上不含外源载体元件的所需细胞类型，其中附加型表达载体用于表达分化编程因子。

公开(公告)号：WO2009076524A3

公开(公告)日：2009-09-11

申请号：PCT/US2008086409

申请日：2008-12-11

Applicant: UNIV NORTH CAROLINA ,  KAFRI TAL

Inventor： KAFRI TAL

IPC: C12N15/867 ,  C12N15/09 ,  C12N15/48 ,  C12N15/49

CPC classification number: C12N15/86 ,  C12N2740/15043 ,  C12N2740/16043 ,  C12N2800/108 ,  C12N2800/24

Abstract: An integration-defective retroviral vector transfer cassette lacking a functional polypurine tract (PPT) is provided. Also provided are isolated nucleic acids that include a heterologous nucleotide sequence, one or two retroviral long terminal repeats (LTRs), a packaging signal, a rev responsive element, and a eukaryotic promoter, wherein the nucleic acid lacks a functional PPT; vectors that include the disclosed isolated nucleic acids; recombinant retroviral particles and mRNAs thereof; retroviral vector kits; and methods for producing integration-defective vector particles, achieving gene expression of a nucleotide sequence of interest, and inserting a nucleotide sequence of interest into a host cell genome in a site-specific or non-specific manner.

Abstract translation: 提供缺乏功能性聚嘌呤道（PPT）的整合缺陷型逆转录病毒载体转移盒。 还提供了包含异源核苷酸序列，一个或两个逆转录病毒长末端重复序列（LTR），包装信号，rev应答元件和真核启动子的分离的核酸，其中所述核酸缺乏功能性PPT; 包括公开的分离的核酸的载体; 重组逆转录病毒颗粒及其mRNA; 逆转录病毒载体试剂盒; 以及用于产生整合缺陷型载体颗粒，实现感兴趣的核苷酸序列的基因表达以及以位点特异性或非特异性方式将感兴趣的核苷酸序列插入宿主细胞基因组的方法。

公开(公告)号：WO2007150036A2

公开(公告)日：2007-12-27

申请号：PCT/US2007/071911

申请日：2007-06-22

Applicant: UNIVERSITY OF MEDICINE & DENTISTRY OF NEW JERSEY ,  GUERRA, Cesar, E. ,  OZER, Harvey, L.

Inventor： GUERRA, Cesar, E. ,  OZER, Harvey, L.

IPC: C12N15/74

CPC classification number: C12N15/85 ,  C12N2800/108 ,  C12N2820/60

Abstract: The invention relates to a vector comprising an origin of replication for episomal maintenance in a metazoan cell, a gene for episomal maintenance in the metazoan cell, and a telomeric polynucleotide sequence. The vector can be used for episomal expression of RNA and polypeptides in metazoan cells.

Abstract translation: 本发明涉及一种载体，其包含后生动物细胞中的附加型维持的复制起点，后生动物细胞中的游离体维持基因和端粒多核苷酸序列。 该载体可用于后生动物细胞中RNA和多肽的附加型表达。

公开(公告)号：WO2007047759A1

公开(公告)日：2007-04-26

申请号：PCT/US2006/040713

申请日：2006-10-17

Applicant: THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA ,  ROBERTSON, Erle, S. ,  VERMA, Subhash, C.

Inventor： ROBERTSON, Erle, S. ,  VERMA, Subhash, C.

IPC: C12Q1/68

CPC classification number: C12N15/85 ,  C12N15/113 ,  C12N2310/14 ,  C12N2710/16411 ,  C12N2800/108 ,  C12N2820/60 ,  C12Q1/702

Abstract: This invention provides methods of reducing a replication of a gammaherpesvirus genome, treating a KSHV infection, and treating or reducing an incidence of a KSHV-associated disease, comprising contacting a subject with a composition that inhibits initiation of DNA replication from a region of a genome of a gammaherpesvirus. The invention also provides isolated DNA molecules capable of episomal replication in a eukaryotic cell, and methods of delivering a recombinant protein or therapeutic RNA molecule, comprising same.

Abstract translation: 本发明提供减少伽pes病毒基因组复制，治疗KSHV感染以及治疗或降低KSHV相关疾病发病率的方法，其包括使受试者与抑制基因组区域的DNA复制起始的组合物接触 的gamma病毒。 本发明还提供能够在真核细胞中附加型复制的分离的DNA分子，以及递送包含其的重组蛋白或治疗性RNA分子的方法。

公开(公告)号：WO2006060769A3

公开(公告)日：2007-02-15

申请号：PCT/US2005043922

申请日：2005-12-05

Applicant: XOMA TECHNOLOGY LTD ,  HANDA MASAHISA ,  HORWITZ ARNOLD ,  BAUTISTA EDDIE ,  COTTER ROBYN

Inventor： HANDA MASAHISA ,  HORWITZ ARNOLD ,  BAUTISTA EDDIE ,  COTTER ROBYN

IPC: C12N15/869 ,  C12N5/00 ,  C12N15/67

CPC classification number: C07K16/00 ,  C12N7/00 ,  C12N15/85 ,  C12N2710/16221 ,  C12N2710/16243 ,  C12N2800/108 ,  C12N2820/10 ,  C12N2820/55 ,  C12N2820/60 ,  C12N2830/42

Abstract: Recombinant expression vectors are provided comprising a 3' UTR of a light chain and an Epstein-Barr virus origin of replication. Also provided are host cells comprising such vectors and methods of producing recombinant protein with such vectors. Additional methods of producing a recombinant protein involve contacting cells with a first and second vector, each of which encode a different polypeptide chain, and wherein the second vector is present in an amount which is about 1.5 to 2.5 times as much as that of the first vector. Cells also can be transfected with a recombinant transient expression vector encoding a protein and are cultured in a medium in a membrane-enhanced culturing vessel to produce recombinant protein.

Abstract translation: 提供重组表达载体，其包含轻链的3'UTR和Epstein-Barr病毒复制起点。 还提供了包含这种载体的宿主细胞和用这种载体产生重组蛋白的方法。 生产重组蛋白质的其它方法包括使细胞与第一和第二载体接触，第一和第二载体各自编码不同的多肽链，其中第二载体的存在量为第一载体的约1.5至2.5倍 向量。 细胞也可以用编码蛋白质的重组瞬时表达载体转染，并在膜增强培养容器中的培养基中培养以产生重组蛋白。