公开(公告)号：US20240191243A1

公开(公告)日：2024-06-13

申请号：US18286847

申请日：2022-04-21

申请人： University of Pittsburgh - Of the Commonwealth System of Higher Education

发明人： Joseph C. Glorioso, III ,  Justus B. COHEN

IPC分类号： C12N15/64 ,  A61K48/00 ,  C12N7/00 ,  C12N15/86

CPC分类号： C12N15/64 ,  A61K48/0091 ,  C12N7/00 ,  C12N15/86 ,  C12N2710/16622 ,  C12N2710/16643 ,  C12N2710/16652 ,  C12N2830/003

摘要： Disclosed are custom HSV-1 packaging genomes for chromosomal integration into a cell line that is devoid of cis-acting HSV origins of replication (ori) and packaging sequences (pac) normally used for incorporation of the viral genome into virus particles, as well as the use of the resulting packaging cell for amplicon packaging.

公开(公告)号：US07655224B2

公开(公告)日：2010-02-02

申请号：US11546726

申请日：2006-10-12

申请人： Evan Y. Snyder ,  Xandra O. Breakefield ,  Karen S. Aboody ,  Ulrich Herrlinger ,  William P. Lynch

发明人： Evan Y. Snyder ,  Xandra O. Breakefield ,  Karen S. Aboody ,  Ulrich Herrlinger ,  William P. Lynch

IPC分类号： A61K48/00 ,  C12N15/63 ,  C12N5/08 ,  A61K31/70

CPC分类号： C12N5/0623 ,  A61K31/4412 ,  A61K31/522 ,  A61K35/763 ,  A61K38/00 ,  A61K48/0075 ,  C12N7/00 ,  C12N15/86 ,  C12N2501/06 ,  C12N2501/999 ,  C12N2510/00 ,  C12N2710/16632 ,  C12N2710/16643 ,  C12N2710/16652 ,  Y02A50/473 ,  A61K2300/00

摘要： The present invention is based upon a surprising finding that stem cells, more particularly neural stem cells, can migrate throughout a brain tumor and track metastatic brain tumor cells. The invention provides a method for treating brain tumors by administering genetically engineered neural stem cells in an individual affected by brain tumors. The invention also provides a method of preparing genetically engineered neural stem cells and a composition comprising genetically engineered neural stem cells in a pharmaceutically acceptable carrier.

摘要翻译： 本发明基于令人惊奇的发现，即干细胞，更具体地说，神经干细胞可以遍及整个脑肿瘤迁移并跟踪转移性脑肿瘤细胞。 本发明提供了通过在受脑肿瘤影响的个体中施用基因工程神经干细胞来治疗脑肿瘤的方法。 本发明还提供了一种制备遗传工程化神经干细胞的方法和包含在药学上可接受的载体中的遗传工程化神经干细胞的组合物。

公开(公告)号：US20020045261A1

公开(公告)日：2002-04-18

申请号：US09795675

申请日：2001-02-28

发明人： Evan Y. Snyder ,  Xandra O. Breakefield ,  Karen S. Aboody ,  Ulrich Herrlinger ,  William P. Lynch

IPC分类号： A61K048/00 ,  C12N005/08

CPC分类号： C12N5/0623 ,  A61K31/4412 ,  A61K31/522 ,  A61K35/763 ,  A61K38/00 ,  A61K48/0075 ,  C12N7/00 ,  C12N15/86 ,  C12N2501/06 ,  C12N2501/999 ,  C12N2510/00 ,  C12N2710/16632 ,  C12N2710/16643 ,  C12N2710/16652 ,  Y02A50/473 ,  A61K2300/00

摘要： The present invention is based upon a surprising finding that stem cells, more particularly neural stem cells, can migrate throughout a brain tumor and track metastatic brain tumor cells. The invention provides a method for treating brain tumors by administering genetically engineered neural stem cells in an individual affected by brain tumors. The invention also provides a method of preparing genetically engineered neural stem cells and a composition comprising genetically engineered neural stem cells in a pharmaceutically acceptable carrier.

摘要翻译： 本发明基于令人惊奇的发现，即干细胞，更具体地说，神经干细胞可以遍及整个脑肿瘤迁移并跟踪转移性脑肿瘤细胞。 本发明提供了通过在受脑肿瘤影响的个体中施用基因工程神经干细胞来治疗脑肿瘤的方法。 本发明还提供了一种制备遗传工程化神经干细胞的方法和包含在药学上可接受的载体中的遗传工程化神经干细胞的组合物。

公开(公告)号：US5288641A

公开(公告)日：1994-02-22

申请号：US923015

申请日：1992-07-30

申请人： Bernard Roizman

发明人： Bernard Roizman

IPC分类号： C07K14/02 ,  C12N15/869 ,  C12N15/86 ,  C12N15/09 ,  C12P21/00

CPC分类号： C12N15/86 ,  C12N2710/16622 ,  C12N2710/16643 ,  C12N2710/16652

摘要： A foreign gene is inserted into a viral genome under the control of promoter-regulatory regions of the genome, thus providing a vector for the expression of the foreign gene. DNA constructs, plasmid vectors containing the constructs useful for expression of the foreign gene, recombinant viruses produced with the vector, and associated methods are disclosed.

摘要翻译： 外源基因在基因组的启动子调控区的控制下插入到病毒基因组中，从而提供外源基因表达的载体。 公开了DNA构建体，含有用于表达外源基因的构建体的质粒载体，用该载体产生的重组病毒和相关方法。

公开(公告)号：US11987805B2

公开(公告)日：2024-05-21

申请号：US17484476

申请日：2021-09-24

申请人： Albert Einstein College of Medicine

发明人： William R. Jacobs, Jr. ,  Lawrence Leung ,  Regy Lukose ,  Anna Paula de Oliveira

IPC分类号： C12N15/01 ,  C07K14/35 ,  C12N7/00 ,  C12N9/22 ,  C12N15/09 ,  C12N15/11 ,  C12N15/90 ,  C12Q1/70

CPC分类号： C12N15/907 ,  C07K14/35 ,  C12N7/00 ,  C12N9/22 ,  C12N15/11 ,  C12Q1/70 ,  C12N2310/20 ,  C12N2710/16652 ,  C12N2800/80

摘要： A genetically modified mammalian cell and genetically modified mammalian cell line comprise a recombination sequence inserted in a target locus on a chromosome of the mammalian cell genome, wherein the recombination sequence comprises Bxb1attB sequence from Mycobacterium smegmatis. A transgenic mammalian cell and transgenic mammalian cell line comprise a heterologous nucleic acid stably integrated in a target locus on a chromosome of the mammalian genome, wherein the heterologous nucleic comprises a heterologous gene configured for expression by the transgenic mammalian cell.

公开(公告)号：US09616119B2

公开(公告)日：2017-04-11

申请号：US14667512

申请日：2015-03-24

申请人： University of Kansas ,  Saint Louis University

发明人： David J. Davido ,  Lynda Anne Morrison

IPC分类号： A61K39/245 ,  A61K39/12 ,  C07K14/005 ,  C12N7/00 ,  A61K39/00

CPC分类号： A61K39/245 ,  A61K39/12 ,  A61K2039/5254 ,  A61K2039/545 ,  C07K14/005 ,  C12N7/00 ,  C12N2710/16621 ,  C12N2710/16622 ,  C12N2710/16634 ,  C12N2710/16652 ,  C12N2710/16662 ,  C12N2710/16671 ,  A61K35/763 ,  A61K2039/53 ,  A61K35/76 ,  C12N2710/16632 ,  C12N2710/16643 ,  C07K14/035

摘要： A mutant HSV-1 (referred to herein as KOS-NA) was generated. KOS-NA contains novel mutations in the UL39 gene, which encodes for a protein that is a large subunit of ribonucleotide reductase (i.e., ICP6). These UL39 mutations were found to alter two amino acids in ICP6 (R950H and L393P) and are responsible for attenuation of KOS-NA in vivo, and resulted in diminished ICP6 protein levels. These novel UL39 mutations regulate the expression and/or stability of ICP6 and severely impact HSV-1 pathogenesis. Mutant HSV viruses containing these mutations appear to protect against HSV infection and can serve as therapeutic vaccines to help combat preexisting HSV infection in infected individuals.

公开(公告)号：US20070280963A1

公开(公告)日：2007-12-06

申请号：US10556885

申请日：2004-05-17

申请人： Alberto Epstein ,  Cecile Zaupa ,  Carine Logvinoff ,  Patrick Lomonte ,  Joelle Thomas

发明人： Alberto Epstein ,  Cecile Zaupa ,  Carine Logvinoff ,  Patrick Lomonte ,  Joelle Thomas

IPC分类号： A61K39/245 ,  A61K31/7088 ,  A61K48/00 ,  A61P31/22 ,  C07H21/00 ,  C12N1/00 ,  C12N15/64 ,  C12N7/04

CPC分类号： C12N7/00 ,  A61K48/00 ,  C12N15/86 ,  C12N2310/122 ,  C12N2710/16643 ,  C12N2710/16652 ,  C12N2710/16662 ,  C12N2800/30 ,  C12N2830/00 ,  C12N2830/002 ,  C12N2840/203

摘要： Improved methods for making non-cytotoxic helper virus-free preparations of herpes virus amplicon vectors or particles, the vectors themselves, recombinant helper virus and cells and methods of using same to treat patients and as tools in therapy and prevention, immunology, molecular biology, biotechnology and genetic engineering. In one embodiment, an amplicon plasmid vector contains at least one transgene that encodes a transgene product which is an interfering RNA molecule that can be converted into a siRNA by the cell machinery.

摘要翻译： 用于制备非细胞毒性辅助病毒无病毒的疱疹病毒扩增子载体或颗粒制剂的方法，载体本身，重组辅助病毒和细胞及其使用方法来治疗患者以及作为治疗和预防，免疫学，分子生物学， 生物技术和遗传工程。 在一个实施方案中，扩增子质粒载体含有编码转基因产物的至少一个转基因，转基因产物是可通过细胞机制转化为siRNA的干扰RNA分子。

公开(公告)号：US5804413A

公开(公告)日：1998-09-08

申请号：US651419

申请日：1996-05-22

申请人： Neal A. DeLuca

发明人： Neal A. DeLuca

IPC分类号： C12N15/09 ,  A61K39/245 ,  A61P31/22 ,  C12N5/10 ,  C12N7/00 ,  C12N15/869 ,  C12N5/06 ,  C12N7/04 ,  C12N15/86

CPC分类号： C12N15/86 ,  C12N7/00 ,  C12N2710/16643 ,  C12N2710/16652

摘要： Cell lines that express complementing levels of herpes simplex virus (HSV) essential immediate early proteins ICP4 and ICP27 as well as ICP4, ICP27 and ICP0 and a method of producing the novel cell lines are disclosed. These cell lines are utilized to provide HSV strains deficient for both (a) ICP4 and ICP27; (b) ICP4, ICP27, ICP22; (c) ICP4, ICP27, ICP0; and, (d) ICP4, ICP27, ICP22 and ICP0, and their generation, and HSV strains deficient for (a) ICP4 and ICP27; (b) ICP4, ICP27, ICP22; (c) ICP4, ICP27, ICP0; and, (d) ICP4, ICP27, ICP22 and ICP0, and one or more additional genes, and their generation. Vectors are provided from these methods of using these HSV strains for gene transfer and for producing site-specific homologous recombination with cellular DNA.

摘要翻译： 公开了表达单纯疱疹病毒（HSV）必需的即时早期蛋白质ICP4和ICP27以及ICP4，ICP27和ICP0的补充水平的细胞系，以及生产新型细胞系的方法。 这些细胞系用于提供（a）ICP4和ICP27缺失的HSV毒株; （b）第4号文件，ICP27，ICP22; （c）第4号，第27号，ICP0; 和（d）ICP4，ICP27，ICP22和ICP0及其产生和缺乏（a）ICP4和ICP27的HSV毒株; （b）第4号文件，ICP27，ICP22; （c）第4号，第27号，ICP0; 和（d）ICP4，ICP27，ICP22和ICP0，以及一个或多个其他基因及其产生。 从使用这些HSV菌株进行基因转移和用于与细胞DNA产生位点特异性同源重组的这些方法提供载体。

公开(公告)号：US20240191251A1

公开(公告)日：2024-06-13

申请号：US18285630

申请日：2022-04-05

申请人： Solid Biosciences Inc.

发明人： Peter Pechan ,  Sarath Chandra Mandava

IPC分类号： C12N15/86 ,  C07K14/47

CPC分类号： C12N15/86 ,  C07K14/4716 ,  C12N2710/16643 ,  C12N2710/16652 ,  C12N2750/14122 ,  C12N2750/14144 ,  C12N2830/50

摘要： The invention described herein provides a recombinant replication-defective virus derived from Herpesvirales order, comprising a defective ICP27 gene that impairs or otherwise does not support replication of the HSV, or a functional equivalent gene thereof; a gene-of-interest (GOI) flanked by AAV ITR sequences inserted at, within, or replacing a non-essential or replaceable essential locus of the HSV; and a coding sequence for AAV Rep and Cap proteins inserted at, within or replacing the non-essential or replaceable essential locus of the HSV. The invention also provides production cell lines for such recombinant replication-defective virus, wherein the cell lines have a coding sequence for ICP27 or a functional equivalent thereof, and wherein the coding sequence has no or minimal sequence overlap with the virus characterized by the defective ICP27 gene that impairs or otherwise does not support replication of the HSV. Methods of using such recombinant replication-defective virus and production cell lines are also provided. rAAV produced using such recombinant replication-defective virus and/or production cell lines and methods of making such rAAV are also provided.

公开(公告)号：US20070031391A1

公开(公告)日：2007-02-08

申请号：US11546726

申请日：2006-10-12

申请人： Evan Snyder ,  Xandra Breakefield ,  Karen Aboody ,  Ulrich Herrlinger ,  William Lynch

发明人： Evan Snyder ,  Xandra Breakefield ,  Karen Aboody ,  Ulrich Herrlinger ,  William Lynch

IPC分类号： A61K48/00 ,  C12N5/08

CPC分类号： C12N5/0623 ,  A61K31/4412 ,  A61K31/522 ,  A61K35/763 ,  A61K38/00 ,  A61K48/0075 ,  C12N7/00 ,  C12N15/86 ,  C12N2501/06 ,  C12N2501/999 ,  C12N2510/00 ,  C12N2710/16632 ,  C12N2710/16643 ,  C12N2710/16652 ,  Y02A50/473 ,  A61K2300/00

摘要： The present invention is based upon a surprising finding that stem cells, more particularly neural stem cells, can migrate throughout a brain tumor and track metastatic brain tumor cells. The invention provides a method for treating brain tumors by administering genetically engineered neural stem cells in an individual affected by brain tumors. The invention also provides a method of preparing genetically engineered neural stem cells and a composition comprising genetically engineered neural stem cells in a pharmaceutically acceptable carrier.