公开(公告)号：US5998205A

公开(公告)日：1999-12-07

申请号：US849117

申请日：1997-08-01

申请人： Paul L. Hallenbeck ,  Yung-Nien Chang ,  Yawen L. Chiang

发明人： Paul L. Hallenbeck ,  Yung-Nien Chang ,  Yawen L. Chiang

IPC分类号： C12N15/09 ,  A61K35/76 ,  A61K38/00 ,  A61K38/44 ,  A61K39/23 ,  A61K39/235 ,  A61K39/245 ,  A61K39/29 ,  A61K48/00 ,  A61P35/00 ,  C12N5/08 ,  C12N5/10 ,  C12N15/861 ,  C12N15/00

CPC分类号： C12N15/86 ,  A61K48/0058 ,  A61K48/00 ,  C12N2710/10343 ,  C12N2820/002 ,  C12N2830/002 ,  C12N2830/008 ,  C12N2830/32 ,  C12N2830/85 ,  C12N2840/20

摘要： The invention generally relates to targeted gene therapy using recombinant vectors and particularly adenovirus vectors. The invention specifically relates to replication-conditional vectors and methods for using them. Such vectors are able to selectively replicate in a target tissue to provide a therapeutic benefit from the presence of the vector per se or from heterologous gene products expressed from the vector and distributed throughout the tissue. In such vectors, a gene essential for replication is placed under the control of a heterologous tissue-specific transcriptional regulatory sequence. Thus, replication is conditioned on the presence of a factor(s) that induces transcription or the absence of a factor(s) that ihibits transcription of the gene by means of the transcriptional regulatory sequence with this vector; therefore, a target tissue can be selectively treated.

摘要翻译： PCT No.PCT / US95 / 15455 Sec。 371日期1997年7月1日 102（e）日期1997年7月1日PCT提交1995年11月28日PCT公布。 公开号WO96 / 17053 日期1996年6月6日本发明一般涉及使用重组载体，特别是腺病毒载体的靶向基因治疗。 本发明具体涉及复制条件向量及其使用方法。 这样的载体能够在靶组织中选择性地复制以从载体本身的存在或从载体表达的分布在整个组织中的异源基因产物提供治疗益处。 在这样的载体中，将复制必需的基因置于异源组织特异性转录调控序列的控制之下。 因此，复制是基于诱导转录的因子的存在或不存在通过使用该载体的转录调节序列抑制基因转录的因子的因子; 因此，可以选择性地处理靶组织。

公开(公告)号：US20080213884A1

公开(公告)日：2008-09-04

申请号：US11977902

申请日：2007-10-25

申请人： Paul L. Hallenbeck ,  Yung-Nien Chang ,  Yawen L. Chiang

发明人： Paul L. Hallenbeck ,  Yung-Nien Chang ,  Yawen L. Chiang

IPC分类号： C12N5/06

CPC分类号： C12N15/86 ,  A61K48/00 ,  A61K48/0058 ,  C12N2710/10343 ,  C12N2820/002 ,  C12N2830/002 ,  C12N2830/008 ,  C12N2830/32 ,  C12N2830/85 ,  C12N2840/20

摘要： The invention generally relates to targeted gene therapy using recombinant vectors and particularly adenovirus vectors. The invention specifically relates to replication-conditional vectors and methods for using them. Such vectors are able to selectively replicate in a target tissue to provide a therapeutic benefit from the presence of the vector per se or from heterologous gene products expressed from the vector and distributed throughout the tissue. In such vectors, a gene essential for replication is placed under the control of a heterologous tissue-specific transcriptional regulatory sequence. Thus, replication is conditioned on the presence of a factor(s) that induces transcription or the absence of a factor(s) that inhibits transcription of the gene by means of the transcriptional regulatory sequence with this vector, therefore, a target tissue can be selectively treated.

摘要翻译： 本发明一般涉及使用重组载体，特别是腺病毒载体的靶向基因治疗。 本发明具体涉及复制条件向量及其使用方法。 这样的载体能够在靶组织中选择性地复制以从载体本身的存在或从载体表达的分布在整个组织中的异源基因产物提供治疗益处。 在这样的载体中，将复制必需的基因置于异源组织特异性转录调控序列的控制之下。 因此，复制受到诱导转录的因子的存在或不存在通过该载体通过转录调节序列抑制基因转录的因子的因素，因此，靶组织可以是 选择性处理。

公开(公告)号：US06551587B2

公开(公告)日：2003-04-22

申请号：US09210936

申请日：1998-12-15

申请人： Paul L. Hallenbeck ,  Yung-Nien Chang ,  Yawen L. Chiang

发明人： Paul L. Hallenbeck ,  Yung-Nien Chang ,  Yawen L. Chiang

IPC分类号： H61K4800

CPC分类号： C12N15/86 ,  A61K48/00 ,  A61K48/0058 ,  C12N2710/10343 ,  C12N2820/002 ,  C12N2830/002 ,  C12N2830/008 ,  C12N2830/32 ,  C12N2830/85 ,  C12N2840/20

摘要： The invention generally relates to targeted gene therapy using recombinant vectors and particularly adenovirus vectors. The invention specifically relates to replication-conditional vectors and methods for using them. Such vectors are able to selectively replicate in a target tissue to provide a therapeutic benefit from the presence of the vector per se or from heterologous gene products expressed from the vector and distributed throughout the tissue. In such vectors, a gene essential for replication is placed under the control of a heterologous tissue-specific transcriptional regulatory sequence. Thus, replication is conditioned on the presence of a factor(s) that induces transcription or the absence of a factor(s) that inhibits transcription of the gene by means of the transcriptional regulatory sequence with this vector, therefore, a target tissue can be selectively treated.

摘要翻译： 本发明一般涉及使用重组载体，特别是腺病毒载体的靶向基因治疗。 本发明具体涉及复制条件向量及其使用方法。 这样的载体能够在靶组织中选择性地复制以从载体本身的存在或从载体表达的分布在整个组织中的异源基因产物提供治疗益处。 在这样的载体中，将复制必需的基因置于异源组织特异性转录调控序列的控制之下。 因此，复制受到诱导转录的因子的存在或不存在通过该载体通过转录调节序列抑制基因转录的因子的因素，因此，靶组织可以是 选择性处理。

公开(公告)号：US06638762B1

公开(公告)日：2003-10-28

申请号：US08974391

申请日：1997-11-19

申请人： Yung-Nien Chang ,  Paul L. Hallenbeck ,  Carl M. Hay ,  David A. Stewart

发明人： Yung-Nien Chang ,  Paul L. Hallenbeck ,  Carl M. Hay ,  David A. Stewart

IPC分类号： C12N1500

CPC分类号： C12N15/86 ,  A61K48/00 ,  C12N2710/10343 ,  C12N2830/008 ,  C12N2830/15 ,  C12N2830/30 ,  C12N2830/32 ,  C12N2830/85 ,  C12N2840/20 ,  C12N2840/44

摘要： The invention generally relates to cell-specific expression vectors. It particularly relates to targeted gene therapy using recombinant expression vectors and particularly adenovirus vectors. The invention specifically relates to replication-conditional expression vectors and methods for using them. Such vectors are able to selectively replicate in a target cell or tissue to provide a therapeutic benefit in a tissue from the presence of the vector per se or from one or more heterologous gene products expressed from the vector and distributed throughout the tissue. In such vectors, a gene essential for replication is placed under the control of a heterologous tissue-specific transcriptional regulatory sequence. Thus, replication is conditioned on the presence of a factor(s) that induces transcription or the absence of a factor(s) that inhibits transcription of the gene by means of the transcriptional regulatory sequence with this vector; therefore, a target tissue can be selectively treated.

摘要翻译： 本发明一般涉及细胞特异性表达载体。 它特别涉及使用重组表达载体，特别是腺病毒载体的靶向基因治疗。 本发明具体涉及复制条件表达载体及其使用方法。 这样的载体能够在靶细胞或组织中选择性复制，以从载体本身的存在或从载体表达的一种或多种异源基因产物提供组织中的治疗益处并且分布在整个组织中。 在这样的载体中，将复制必需的基因置于异源组织特异性转录调控序列的控制之下。 因此，复制受到诱导转录或不存在通过与该载体的转录调控序列抑制基因转录的因子的因素的制约; 因此，可以选择性地处理靶组织。

公开(公告)号：US20080076172A1

公开(公告)日：2008-03-27

申请号：US11977533

申请日：2007-10-24

申请人： Paul Hallenbeck ,  Yung-Nien Chang ,  Yawen Chiang

发明人： Paul Hallenbeck ,  Yung-Nien Chang ,  Yawen Chiang

IPC分类号： C12N15/00

CPC分类号： C12N15/86 ,  A61K48/00 ,  A61K48/0058 ,  C12N2710/10343 ,  C12N2820/002 ,  C12N2830/002 ,  C12N2830/008 ,  C12N2830/32 ,  C12N2830/85 ,  C12N2840/20

摘要： The invention generally relates to targeted gene therapy using recombinant vectors and particularly adenovirus vectors. The invention specifically relates to replication-conditional vectors and methods for using them. Such vectors are able to selectively replicate in a target tissue to provide a therapeutic benefit from the presence of the vector per se or from heterologous gene products expressed from the vector and distributed throughout the tissue. In such vectors, a gene essential for replication is placed under the control of a heterologous tissue-specific transcriptional regulatory sequence. Thus, replication is conditioned on the presence of a factor(s) that induces transcription or the absence of a factor(s) that inhibits trancription of the gene by means of the transcriptional regulatory sequence with this vector; therefore, a target tissue can be selectively treated.

摘要翻译： 本发明一般涉及使用重组载体，特别是腺病毒载体的靶向基因治疗。 本发明具体涉及复制条件向量及其使用方法。 这样的载体能够在靶组织中选择性地复制以从载体本身的存在或从载体表达的分布在整个组织中的异源基因产物提供治疗益处。 在这样的载体中，将复制必需的基因置于异源组织特异性转录调控序列的控制之下。 因此，复制受到诱导转录或不存在通过用该载体通过转录调控序列抑制基因转录的因子的因子的影响; 因此，可以选择性地处理靶组织。

公开(公告)号：US06627442B1

公开(公告)日：2003-09-30

申请号：US09653088

申请日：2000-08-31

申请人： Laurent Humeau ,  Wei Han ,  Xiaobin Lu ,  Vladimir Slepushkin ,  Mechelle Lesher ,  Brian Davis ,  Yung-Nien Chang ,  Boro Dropulic

发明人： Laurent Humeau ,  Wei Han ,  Xiaobin Lu ,  Vladimir Slepushkin ,  Mechelle Lesher ,  Brian Davis ,  Yung-Nien Chang ,  Boro Dropulic

IPC分类号： C12N1585

CPC分类号： C12N15/86 ,  A61K48/00 ,  C12N2740/16043

摘要： The present invention provides methods, as well as compositions related thereto, for the efficient transduction of cells using viral vectors. The efficiency of transduction is increased by contacting the cell to be transduced with one or more molecules that bind the cell surface. Contact with a cell surface binding molecule may occur before, after, or simultaneously with contact between the viral vector and the cell. The transduced vectors may be constructed to express a gene of interest, permitting the transduced cells to be used as therapeutic and prophylactic agents.

摘要翻译： 本发明提供了使用病毒载体有效转导细胞的方法以及与之相关的组合物。 通过使待转导的细胞与结合细胞表面的一种或多种分子接触来增加转导效率。 与细胞表面结合分子的接触可以在病毒载体和细胞之间的接触之前，之后或同时发生。 可以构建转导载体以表达感兴趣的基因，允许转导的细胞用作治疗和预防剂。

公开(公告)号：US08420352B2

公开(公告)日：2013-04-16

申请号：US12870782

申请日：2010-08-27

申请人： George A. Oyler ,  Yung-Nien Chang

发明人： George A. Oyler ,  Yung-Nien Chang

IPC分类号： C07K14/33 ,  C12N1/20 ,  C12N15/62 ,  C12P21/02

CPC分类号： C12N5/0696 ,  A61K35/74 ,  A61K38/164 ,  C07K14/33 ,  C07K14/4702 ,  C07K2319/00 ,  C07K2319/10 ,  C07K2319/55 ,  C12N5/00 ,  C12N2501/602 ,  C12N2501/603

摘要： A novel protein delivery system to generate induced pluripotent stem (iPS) cells is described. The delivery system comprises a construct with a receptor binding domain that recognizes a receptor in a somatic cell, a translocation domain that allows the transfer of an inducer into the cytosolic space, and a cargo bearing domain to which the inducer is attached and facilitates transfer of the inducer into the cell.

摘要翻译： 描述了产生诱导多能干细胞（iPS）细胞的新型蛋白质递送系统。 递送系统包括具有受体结合结构域的构建体，其识别体细胞中的受体，允许诱导物转移到细胞溶质空间中的易位结构域，以及引诱物附着的携带轴承结构域，并促进 诱导剂进入细胞。

公开(公告)号：US20060121579A1

公开(公告)日：2006-06-08

申请号：US11011264

申请日：2004-12-13

申请人： Yung-Nien Chang ,  Yajin Ni ,  Boro Dropulic

发明人： Yung-Nien Chang ,  Yajin Ni ,  Boro Dropulic

IPC分类号： C12Q1/68 ,  C12P19/34 ,  C12N5/00 ,  C12N5/02

CPC分类号： C12N7/00 ,  C12N15/635 ,  C12N2740/16052 ,  C12N2740/16122 ,  C12N2740/16222 ,  C12N2740/16322

摘要： The present invention provides nucleic acid constructs, expression systems, and methods relating to the regulation of gene expression. The invention may be applied to regulate the expression of any coding sequence of interest, including those coding for viral components necessary for the packaging of viral particles.

公开(公告)号：US20080103109A1

公开(公告)日：2008-05-01

申请号：US11977903

申请日：2007-10-25

申请人： Paul Hallenbeck ,  Yung-Nien Chang ,  Yawen Chiang

发明人： Paul Hallenbeck ,  Yung-Nien Chang ,  Yawen Chiang

IPC分类号： A61K31/70 ,  A61P43/00

CPC分类号： C12N15/86 ,  A61K48/00 ,  A61K48/0058 ,  C12N2710/10343 ,  C12N2820/002 ,  C12N2830/002 ,  C12N2830/008 ,  C12N2830/32 ,  C12N2830/85 ,  C12N2840/20

摘要： The invention generally relates to targeted gene therapy using recombinant vectors and particularly adenovirus vectors. The invention specifically relates to replication-conditional vectors and methods for using them. Such vectors are able to selectively replicate in a target tissue to provide a therapeutic benefit from the presence of the vector per se or from heterologous gene products expressed from the vector and distributed throughout the tissue. In such vectors, a gene essential for replication is placed under the control of a heterologous tissue-specific transcriptional regulatory sequence. Thus, replication is conditioned on the presence of a factor(s) that induces transcription or the absence of a factor(s) that inhibits transcription of the gene by means of the transcriptional regulatory sequence with this vector; therefore, a target tissue can be selectively treated.

摘要翻译： 本发明一般涉及使用重组载体，特别是腺病毒载体的靶向基因治疗。 本发明具体涉及复制条件向量及其使用方法。 这样的载体能够在靶组织中选择性地复制以从载体本身的存在或从载体表达的分布在整个组织中的异源基因产物提供治疗益处。 在这样的载体中，将复制必需的基因置于异源组织特异性转录调控序列的控制之下。 因此，复制受到诱导转录或不存在通过与该载体的转录调控序列抑制基因转录的因子的因素的制约; 因此，可以选择性地处理靶组织。

公开(公告)号：US20090305358A1

公开(公告)日：2009-12-10

申请号：US11921842

申请日：2006-07-01

申请人： Yung-Nien Chang

发明人： Yung-Nien Chang

IPC分类号： C12N15/64 ,  C12N15/63 ,  C12N5/10 ,  C12N15/00

CPC分类号： C12N15/86 ,  C12N2710/22043 ,  C12N2800/108 ,  C12N2800/30

摘要： The invention discloses the production of double stranded DNA (dsDNA) vectors capable of delivering nucleic acids, including cDNA, antisense, ribozyme, and small interference RNA into cells. The invention also describes nucleic acid constructs as well as methods for the production of the dsDNA vectors.

摘要翻译： 本发明公开了能够将细胞中包含cDNA，反义，核酶和小干扰RNA的核酸的双链DNA（dsDNA）载体的生产。 本发明还描述了核酸构建体以及生产dsDNA载体的方法。