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1. 发明授权

US08927251B2 Targeting of herpes simplex virus to specific receptors 有权
标题翻译：将单纯疱疹病毒靶向特异性受体
公开(公告)号：US08927251B2
公开(公告)日：2015-01-06
申请号：US12065455
申请日：2006-08-30
申请人： Guoying Zhou , Bernard Roizman
发明人： Guoying Zhou , Bernard Roizman
IPC分类号： C12N7/01 , C12N15/869 , A61K39/245 , A61K49/00 , A61K47/48 , A61K38/20 , C12Q1/70
CPC分类号： C07K16/18 , A61K35/00 , A61K38/2086 , A61K47/642 , A61K47/6801 , A61K47/69 , A61K47/6901 , C07K14/5437 , C12N15/86 , C12N15/8695 , C12N2710/16632 , C12N2710/16643 , C12N2710/16645 , C12N2799/028 , C12N2810/50 , C12N2810/852 , C12Q1/705
摘要： The invention relates to engineered Herpes simplex virus (HSV) particles that are targeted to one or more specific binding pair members, such as receptors. Also, recombinant vectors for producing such HSV particles are provided. By reducing the affinity of HSV for its natural receptor(s) and increasing the affinity for a selected receptor, the HSV particles of the invention are useful for targeting cells that express the selected receptor, which itself may be a product of genetic engineering. The ability to selectively target cells render the HSV particles. particularly useful in selectively diagnosing, treating, and imaging cells bearing the selected binding pair member, such as a receptor. The invention also provides for polynucleotide-based therapy to cells bearing the selected binding pair member such as a receptor.
摘要翻译：本发明涉及靶向一种或多种特异性结合对成员（例如受体）的工程化单纯疱疹病毒（HSV）颗粒。此外，提供了用于生产这种HSV颗粒的重组载体。通过降低HSV对其天然受体的亲合力并增加对选定受体的亲和力，本发明的HSV颗粒可用于靶向表达所选受体的细胞，其本身可能是遗传工程的产物。选择性靶向细胞的能力使HSV颗粒。特别可用于选择性地诊断，治疗和成像携带所选择的结合对成员例如受体的细胞。本发明还提供了携带选定结合对成员例如受体的细胞的基于多核苷酸的治疗。

2. 发明授权

US08802080B2 Raav expression systems for genetic modification of specific capsid proteins 有权
标题翻译：用于特异性衣壳蛋白遗传修饰的Raav表达系统
公开(公告)号：US08802080B2
公开(公告)日：2014-08-12
申请号：US10513059
申请日：2003-05-01
申请人： Kenneth H. Warrington , Shaun R. Opie , Nicholas Muzyczka
发明人： Kenneth H. Warrington , Shaun R. Opie , Nicholas Muzyczka
IPC分类号： C12N15/864 , C12N7/01 , C12N5/10 , C12N15/63 , C07K14/015
CPC分类号： A61K48/005 , C07K14/005 , C12N15/86 , C12N2750/14122 , C12N2750/14143 , C12N2750/14145 , C12N2810/50 , C12N2810/858
摘要： Disclosed are improved recombinant adeno-associated viral (rAAV) vectors having mutations in one or more capsid proteins. Exemplary vectors are provided that have altered affinity for heparin or heparin sulfate, as well as vectors, expression systems, and rAAV virions that lack functional VP2 protein expression, but are nevertheless, fully virulent. Also provided by the invention are rAAV vector-based compositions, virus particles, host cells, and pharmaceutical formulations that comprise them useful in the expression of selected therapeutic proteins, polypeptides, peptides, antisense oligonucleotides and/or ribozymes in selected mammals, including organs, tissues, and human host cells.
摘要翻译：公开了在一种或多种衣壳蛋白中具有突变的改良的重组腺相关病毒（rAAV）载体。提供了对肝素或硫酸肝素具有改变的亲和力的示例性载体，以及缺乏功能性VP2蛋白表达但仍然是完全毒性的载体，表达系统和rAAV病毒体。本发明还提供了基于rAAV载体的组合物，病毒颗粒，宿主细胞和药物制剂，其包含它们可用于选择的哺乳动物（包括器官）中选择的治疗性蛋白质，多肽，肽，反义寡核苷酸和/或核酶的表达，组织和人宿主细胞。

3. 发明授权

US08652460B2 Gene delivery system and method of use 有权
标题翻译：基因递送系统及使用方法
公开(公告)号：US08652460B2
公开(公告)日：2014-02-18
申请号：US13290989
申请日：2011-11-07
申请人： Noriyuki Kasahara , Christopher R. Logg , W. French Anderson
发明人： Noriyuki Kasahara , Christopher R. Logg , W. French Anderson
IPC分类号： A61K48/00 , C12N15/00
CPC分类号： C12N15/86 , C12N2740/13043 , C12N2740/13045 , C12N2810/50 , C12N2810/80 , C12N2810/854 , C12N2810/859 , C12N2830/002 , C12N2830/008 , C12N2830/15 , C12N2830/85 , C12N2840/20 , C12N2840/203 , C12N2840/85
摘要： A recombinant replication competent retrovirus for gene deliver and gene therapy is provided. The recombinant retrovirus has a heterologous nucleic acid sequence, a sequence encoding a cell- or tissue-specific ligand or a sequence for transcriptional targeting, or a combination of both a cell- or tissue-specific ligand and a cell- or tissue-specific transcriptional targeting sequence.
摘要翻译：提供了用于基因递送和基因治疗的重组复制型逆转录病毒。重组逆转录病毒具有异源核酸序列，编码细胞或组织特异性配体的序列或用于转录靶向的序列，或细胞或组织特异性配体和细胞或组织特异性转录的组合目标序列。

4. 发明申请

US20100086610A1 VAULT AND VAULT-LIKE CARRIER MOLECULES 有权
标题翻译：缬氨酸和缬氨酸样载体分子
公开(公告)号：US20100086610A1
公开(公告)日：2010-04-08
申请号：US12252200
申请日：2008-10-15
申请人： Leonard H. Rome , Valerie A. Kickhoefer , Sujna Raval-Fernandes , Phoebe L. Stewart
发明人： Leonard H. Rome , Valerie A. Kickhoefer , Sujna Raval-Fernandes , Phoebe L. Stewart
IPC分类号： A61K9/14 , A61K38/02 , A61K38/43 , A61K31/7088
CPC分类号： C12N15/86 , A61K38/00 , A61K38/1709 , A61K38/45 , A61K38/465 , A61K47/42 , A61K47/6901 , A61K49/0002 , A61K49/0056 , B82Y5/00 , C07K14/47 , C07K2319/00 , C12N9/1077 , C12N9/16 , C12N15/88 , C12N2710/14042 , C12N2810/50 , C12Y204/02003 , C12Y301/03067 , G01N33/5005 , G01N33/58 , G01N33/582
摘要： A method of using vaults as carrier molecules to deliver one or more than one substance to an organism, or to a specific tissue or to specific cells, or to an environmental medium. A vault-like particle. A method of preventing damage by one or more than one substance to an organism, to a specific tissue, to specific cells, or to an environmental medium, by sequestering the one or more than one substance within a vault-like particle. A method of delivering one or more than one substance or a sensor to an organism, to a specific tissue, to specific cells, or to an environmental medium. According to another embodiment of the present invention, there is provided a method of making vault-like particles, and making vault-like particles comprising one or more than one substance, or one or more than one sensor.
摘要翻译：使用穹顶作为载体分子将一种或多种物质递送到生物体，或特定组织或特定细胞或环境介质的方法。穹顶状粒子。通过将一种或多于一种的物质隔离在拱顶状颗粒内，防止一种或多于一种物质对生物，特定组织，特定细胞或环境介质的损伤的方法。将一种或多种物质或传感器递送到生物体，特定组织，特定细胞或环境介质的方法。根据本发明的另一个实施方案，提供了制备穹顶状颗粒并制备包含一种或多于一种物质或一种或多于一种传感器的穹顶状颗粒的方法。

5. 发明申请

US20090286321A1 METHODS FOR TARGETING MODIFIED RAAV VECTORS TO MAMMALIAN CELLS 审中-公开
标题翻译：将修饰的RAAV载体定向到马马利亚细胞的方法
公开(公告)号：US20090286321A1
公开(公告)日：2009-11-19
申请号：US12363498
申请日：2009-01-30
申请人： Kenneth H. Warrington , Shaun R. Opie , Nicholas Muzyczka
发明人： Kenneth H. Warrington , Shaun R. Opie , Nicholas Muzyczka
IPC分类号： C12N15/861
CPC分类号： C12N15/86 , A61K48/00 , C12N2750/14143 , C12N2750/14145 , C12N2810/50
摘要： Disclosed are improved VP2-modified recombinant adeno-associated viral (rAAV) vectors, expression systems, and rAAV virions that are fully virulent, yet lack functional VP2 protein expression. Also disclosed are pharmaceutical compositions, virus particles, host cells, and pharmaceutical formulations that comprise these modified vectors useful in the expression of therapeutic proteins, polypeptides, peptides, antisense oligonucleotides and/or ribozymes in the cells and tissues of selected mammals, including, for example, human tissues and host cells.
摘要翻译：公开了改进的VP2修饰的重组腺相关病毒（rAAV）载体，表达系统和完全毒力但缺乏功能性VP2蛋白表达的rAAV病毒粒子。还公开了包含这些修饰的载体的药物组合物，病毒颗粒，宿主细胞和药物制剂，其用于在所选哺乳动物的细胞和组织中表达治疗性蛋白质，多肽，肽，反义寡核苷酸和/或核酶，包括用于例如，人体组织和宿主细胞。

6. 发明申请

US20090215879A1 METHODS AND COMPOSITIONS FOR ADENO-ASSOCIATED VIRUS (AAV) WITH HI LOOP MUTATIONS 审中-公开
标题翻译：用于具有HI循环突变的腺相关病毒（AAV）的方法和组合物
公开(公告)号：US20090215879A1
公开(公告)日：2009-08-27
申请号：US12369945
申请日：2009-02-12
申请人： NINA DIPRIMIO , RICHARD JUDE SAMULSKI
发明人： NINA DIPRIMIO , RICHARD JUDE SAMULSKI
IPC分类号： A61K31/7088 , C07K14/00 , C12N15/00 , C12N15/87
CPC分类号： A61K31/7088 , A61K2039/525 , C07K14/005 , C12N2750/14122 , C12N2810/50
摘要： The invention provides modified AAV capsid proteins comprising substitutions in the HI loop. Suitable substitutions include affinity tags, sequences that facilitate detection and/or targeting peptides. The invention also provides virus capsids and virus vectors comprising the modified AAV capsid proteins and methods of using the same. Further provided are methods of purifying the modified AAV capsid subunits, virus capsids and virus vectors of the invention.
摘要翻译：本发明提供在HI环中包含取代的修饰的AAV衣壳蛋白。合适的取代包括亲和标签，促进检测和/或靶向肽的序列。本发明还提供了包含经修饰的AAV衣壳蛋白的病毒衣壳和病毒载体及其使用方法。还提供纯化本发明的修饰的AAV衣壳亚单位，病毒衣壳和病毒载体的方法。

7. 发明申请

US20090148411A1 VIRAL VECTOR-BASED GENE THERAPY METHODS AND COMPOSITIONS 审中-公开
标题翻译：基于载体的基因治疗方法和组合物
公开(公告)号：US20090148411A1
公开(公告)日：2009-06-11
申请号：US12363454
申请日：2009-01-30
申请人： Kenneth H. Warrington , Shaun R. Opie , Nicholas Muzyczka
发明人： Kenneth H. Warrington , Shaun R. Opie , Nicholas Muzyczka
IPC分类号： A61K35/76 , A61K31/7088
CPC分类号： C12N15/86 , A61K48/00 , C12N2750/14143 , C12N2750/14145 , C12N2810/50
摘要： Disclosed are improved VP2-modified recombinant adeno-associated viral (rAAV) vectors, expression systems, and rAAV virions that are fully virulent, yet lack functional VP2 protein expression. Also disclosed are pharmaceutical compositions, virus particles, host cells, and pharmaceutical formulations that comprise these modified vectors useful in the expression of therapeutic proteins, polypeptides, peptides, antisense oligonucleotides and/or ribozymes in the cells and tissues of selected mammals, including, for example, human tissues and host cells.
摘要翻译：公开了改进的VP2修饰的重组腺相关病毒（rAAV）载体，表达系统和完全毒力但缺乏功能性VP2蛋白表达的rAAV病毒粒子。还公开了包含这些修饰的载体的药物组合物，病毒颗粒，宿主细胞和药物制剂，其用于在所选哺乳动物的细胞和组织中表达治疗性蛋白质，多肽，肽，反义寡核苷酸和/或核酶，包括用于例如，人体组织和宿主细胞。

8. 发明申请

US20080241906A1 Scleroprotein of an adeno-associated virus with modified chromatographic properties, the production thereof and use of the same 审中-公开
标题翻译：具有改良色谱性质的腺相关病毒的硬化蛋白，其生产和使用
公开(公告)号：US20080241906A1
公开(公告)日：2008-10-02
申请号：US11903662
申请日：2007-09-24
申请人： Michael Hallek , Anne Girod , Martin Ried , Christof Korner , Ulrich Moebius
发明人： Michael Hallek , Anne Girod , Martin Ried , Christof Korner , Ulrich Moebius
IPC分类号： C12N7/04 , C12N7/02
CPC分类号： C12N15/86 , A61K48/00 , C07K14/005 , C12N2750/14122 , C12N2750/14143 , C12N2750/14145 , C12N2810/50
摘要： The invention relates to a scleroprotein of an adeno-associated virus which contains at least one mutation. Said mutation causes the chromatographic properties to be modified. The invention also relates to the production of said scleroprotein and the use thereof.
摘要翻译：本发明涉及含有至少一个突变的腺相关病毒的硬皮蛋白。所述突变导致色谱性质被修饰。本发明还涉及所述硬皮蛋白的产生及其用途。

9. 发明申请

US20070087045A1 Lipid carrier and method of preparing the same 审中-公开
标题翻译：脂质载体及其制备方法
公开(公告)号：US20070087045A1
公开(公告)日：2007-04-19
申请号：US11249326
申请日：2005-10-14
申请人： Shyh-Dar Li , Ae-June Wang
发明人： Shyh-Dar Li , Ae-June Wang
IPC分类号： A61K48/00 , A61K9/127 , C12N15/88
CPC分类号： C12N15/88 , A61K9/1271 , A61K9/1272 , C12N2810/50
摘要： A lipid carrier. The carrier includes a lipid based particle comprising a cationic lipid, a cholesterol, a neutral phospholipid, and a neutral lipid, wherein the cationic lipid is about 100 parts by weight, the cholesterol is about 25˜100 parts by weight, the neutral phospholipid is about 25˜100 parts by weight, and the neutral lipid is about 25˜150 parts by weight. The invention also provides a method of preparing the lipid carrier.
摘要翻译：脂质载体。载体包括包含阳离子脂质，胆固醇，中性磷脂和中性脂质的脂质颗粒，其中阳离子脂质为约100重量份，胆固醇为约25〜100重量份，中性磷脂为约25〜100重量份，中性脂质为约25〜150重量份。本发明还提供了制备脂质载体的方法。

10. 发明申请

US20060183703A1 Circular expression construct for gene therapeutic applications 审中-公开
标题翻译：基因治疗应用的循环表达构建体
公开(公告)号：US20060183703A1
公开(公告)日：2006-08-17
申请号：US11299327
申请日：2005-12-09
申请人： Matthias Schroff , Colin Smith
发明人： Matthias Schroff , Colin Smith
IPC分类号： A61K48/00 , C12P19/34
CPC分类号： A61K48/005 , A61K48/00 , A61K2039/53 , C07K14/005 , C12N15/85 , C12N15/88 , C12N2730/10122 , C12N2810/40 , C12N2810/50 , C12P19/34
摘要： Method for producing a circular minimalist expression construct closed in an annular manner, from a double-strand DNA, an expression construct produced according to said method, and the use of the same in gene therapy and vaccination. The abstract of the disclosure is submitted herewith as required by 37 C.F.R. §1.72(b). As stated in 37 C.F.R. §1.72(b): A brief abstract of the technical disclosure in the specification must commence on a separate sheet, preferably following the claims, under the heading “Abstract of the Disclosure.” The purpose of the abstract is to enable the Patent and Trademark Office and the public generally to determine quickly from a cursory inspection the nature and gist of the technical disclosure. The abstract shall not be used for interpreting the scope of the claims. Therefore, any statements made relating to the abstract are not intended to limit the claims in any manner and should not be interpreted as limiting the claims in any manner.
摘要翻译：从双链DNA，根据所述方法制备的表达构建体以环状方式产生环状极简表达构建体的方法及其在基因治疗和疫苗接种中的应用。披露摘要根据37 C.F.R.的要求提交。 §1.72（b）。如37 C.F.R. §1.72（b）：说明书中技术性披露的简要摘要必须在单独的表格上开始，优选按照权利要求，在“披露摘要”标题下。摘要的目的是使专利商标局和公众一般能够从粗略检查中快速确定技术披露的性质和要点。摘要不得用于解释权利要求的范围。因此，与摘要有关的任何陈述不旨在以任何方式限制权利要求，也不应被解释为以任何方式限制权利要求。

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