公开(公告)号：US07838659B2

公开(公告)日：2010-11-23

申请号：US12036365

申请日：2008-02-25

申请人： Alfred S. Lewin ,  David C. Bloom ,  Gregory S. Schultz ,  Sonal Sanjeev Tuli ,  Jia Liu

发明人： Alfred S. Lewin ,  David C. Bloom ,  Gregory S. Schultz ,  Sonal Sanjeev Tuli ,  Jia Liu

IPC分类号： C07H21/04

CPC分类号： C07K14/005 ,  C12N15/1133 ,  C12N2310/111 ,  C12N2310/121 ,  C12N2510/02 ,  C12N2710/16622

摘要： Hammerhead ribozymes that target components critical to HSV replication (ICP4, UL20, UL30, and UL54) were synthesized and shown to efficiently cleave target RNA encoding a portion of these components in in vitro assays. Several cloned cell lines stably expressing these ribozymes were established. HSV-mediated plaque formation was dramatically reduced in cells stably transfected with an anti-HSV ribozyme compared to non-transfected cells.

摘要翻译： 合成针对HSV复制（ICP4，UL20，UL30和UL54）关键组分的锤头核酶，并显示出在体外测定中有效切割编码这些成分的一部分的靶RNA。 建立了稳定表达这些核酶的几种克隆细胞系。 与未转染细胞相比，用抗HSV核酶稳定转染的细胞中HSV介导的斑块形成显着降低。

公开(公告)号：US20090131644A1

公开(公告)日：2009-05-21

申请号：US12036365

申请日：2008-02-25

申请人： Alfred S. Lewin ,  David C. Bloom ,  Gregory S. Schultz ,  Sonal Sanjeev Tuli ,  Jia Liu

发明人： Alfred S. Lewin ,  David C. Bloom ,  Gregory S. Schultz ,  Sonal Sanjeev Tuli ,  Jia Liu

IPC分类号： C07H21/02

CPC分类号： C07K14/005 ,  C12N15/1133 ,  C12N2310/111 ,  C12N2310/121 ,  C12N2510/02 ,  C12N2710/16622

摘要： Hammerhead ribozymes that target components critical to HSV replication (ICP4, UL20, UL30, and UL54) were synthesized and shown to efficiently cleave target RNA encoding a portion of these components in in vitro assays. Several cloned cell lines stably expressing these ribozymes were established. HSV-mediated plaque formation was dramatically reduced in cells stably transfected with an anti-HSV ribozyme compared to non-transfected cells.

摘要翻译： 合成针对HSV复制（ICP4，UL20，UL30和UL54）关键组分的锤头核酶，并显示出在体外测定中有效切割编码这些成分的一部分的靶RNA。 建立了稳定表达这些核酶的几种克隆细胞系。 与未转染细胞相比，用抗HSV核酶稳定转染的细胞中HSV介导的斑块形成显着降低。

公开(公告)号：US5212057A

公开(公告)日：1993-05-18

申请号：US455483

申请日：1989-12-22

申请人： Richard W. Moyer ,  David C. Bloom

发明人： Richard W. Moyer ,  David C. Bloom

IPC分类号： C12N15/863 ,  C12P7/00 ,  C12Q1/70

CPC分类号： C12N15/86 ,  C12P7/00 ,  C12Q1/70 ,  C12N2710/24143 ,  C12N2710/24145

摘要： Disclosed is a novel biological system for 1) the evaluation of potential complications of live virus vaccines prior to actual testing in animals or in the field, and 2) the construction of vaccine vectors in which the ability to grow within specific organs or tissues has selectively been eliminated from the virus.

摘要翻译： 公开了一种新颖的生物系统，用于1）在动物或现场实际测试之前评估活病毒疫苗的潜在并发症，以及2）在特定器官或组织内生长能力有选择性的疫苗载体的构建 被从病毒中消除。

公开(公告)号：US09023617B2

公开(公告)日：2015-05-05

申请号：US10590136

申请日：2005-02-17

申请人： David C. Bloom ,  Antonio L. Amelio

发明人： David C. Bloom ,  Antonio L. Amelio

IPC分类号： C12N15/86 ,  C12N15/85 ,  A61K48/00

CPC分类号： A61K31/713 ,  A61K48/00 ,  C12N15/85 ,  C12N15/86 ,  C12N2710/16611 ,  C12N2710/16622 ,  C12N2710/16633 ,  C12N2710/16642 ,  C12N2710/16643 ,  C12N2830/007 ,  C12N2830/40 ,  C12N2830/60

摘要： Disclosed are genetic expression cassettes, and vector comprising them useful for the delivery of nucleic acid segments encoding selected therapeutic constructs (including for example, peptides, polypeptides, ribozymes, and catalytic RN molecules), to selected cells and tissues of vertebrate animals. The disclosed genetic constructs are useful in the development of gene therapy vectors, including for example, viral vectors such as HSV, retroviral, lentiviral, AV, and rAAV vectors. The expression cassettes disclosed herein provide new tools in the field of gene therapy, and for the treatment of mammalian, and in particular, human diseases, disorders, and dysfunctions. The disclosed compositions may be utilized in a variety of investigative, diagnostic and therapeutic regimens, including the prevention and treatment of a variety of human diseases.

摘要翻译： 公开了遗传表达盒和包含它们的载体，其可用于将编码所选择的治疗构建体（包括例如肽，多肽，核酶和催化性RN分子）的核酸片段递送至脊椎动物的选定细胞和组织。 所公开的遗传构建体可用于开发基因治疗载体，包括例如病毒载体如HSV，逆转录病毒，慢病毒，AV和rAAV载体。 本文公开的表达盒提供了在基因治疗领域和用于治疗哺乳动物，特别是人类疾病，障碍和功能障碍领域的新工具。 所公开的组合物可以用于多种调查，诊断和治疗方案，包括预防和治疗各种人类疾病。