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公开(公告)号：US08865881B2
公开(公告)日：2014-10-21
申请号：US13400945
申请日：2012-02-21
CPC分类号： C07K16/109 , C07K16/1045 , C12N7/00 , C12N2750/14141 , C12N2750/14143
摘要： Disclosed herein are compositions, systems and methods for delivery of proteins of interest using adeno-associated virus (AAV) vectors.
摘要翻译： 本文公开了使用腺相关病毒（AAV）载体递送感兴趣的蛋白质的组合物，系统和方法。
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公开(公告)号：US20130316366A1
公开(公告)日：2013-11-28
申请号：US13901940
申请日：2013-05-24
申请人： Kenneth Yu , David Baltimore , Lili Yang
发明人： Kenneth Yu , David Baltimore , Lili Yang
CPC分类号： C12N15/85 , C07K2319/00 , C12N15/86 , C12N15/907 , C12N2740/15043 , G01N33/582 , G01N33/68
摘要： Some embodiments herein provide compositions and methods for expressing secreted and cell-surface-bound polypeptides in a single cell. In some embodiments, secreted and cell-surface polypeptide are produced from a single polynucleotide. The polynucleotide can comprise a sequence (or sequence encoding a polypeptide) that mediates separation of a membrane anchor from the polypeptide. In some embodiments, a desired ratio of secreted to surface-bound polypeptide is obtained by selecting a sequence that mediates a desired level of separation of the membrane anchor from the polypeptide.
摘要翻译： 本文的一些实施方案提供了用于在单个细胞中表达分泌的和细胞表面结合的多肽的组合物和方法。 在一些实施方案中，分泌的和细胞表面多肽由单个多核苷酸产生。 多核苷酸可以包含介导膜锚定体与多肽分离的序列（或编码多肽的序列）。 在一些实施方案中，分泌到表面结合多肽的期望比例通过选择介导膜锚与多肽分离的期望水平的序列来获得。
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公开(公告)号：US20130065316A1
公开(公告)日：2013-03-14
申请号：US13673056
申请日：2012-11-09
申请人： David Baltimore
发明人： David Baltimore
IPC分类号： G01N35/00
CPC分类号： G01N33/487 , Y10T436/11
摘要： A system and method for analyzing a biological substance, the device comprising: a specimen input device; at least one pumping device in flow communication with a chemical reservoir and said specimen input device; and at least one flow cell in flow communication with said at least one pumping device via at least one flow valve, wherein said at least one flow cell is configured to contain said specimen and includes a sensing device configured to sense at least one characteristic of said specimen.
摘要翻译： 一种用于分析生物物质的系统和方法，所述装置包括：样本输入装置; 至少一个与化学储存器和所述样本输入装置流动连通的抽吸装置; 以及至少一个与所述至少一个泵送装置通过至少一个流量阀流动连通的流动池，其中所述至少一个流动池被配置为容纳所述样本并且包括感测装置，所述感测装置被配置为感测所述 标本。
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4.发明授权公开(公告)号：US08361982B2
公开(公告)日：2013-01-29
申请号：US12769157
申请日：2010-04-28
CPC分类号： C12N15/1132 , A61K48/00 , C12N15/111 , C12N15/113 , C12N15/1138 , C12N15/63 , C12N15/86 , C12N2310/111 , C12N2310/14 , C12N2330/30 , C12N2740/16043 , C12N2740/16045 , C12N2760/20122 , C12N2799/027 , C12N2810/609 , C12N2830/003 , C12N2830/008 , C12N2830/48 , C12N2840/203
摘要： In one aspect, the invention provides methods and compositions for the expression of small RNA molecules within a cell using a retroviral vector (FIG. 1A). Small interfering RNA (siRNA) can be expressed using the methods of the invention within a cell. In a further aspect, the invention provides methods for producing siRNA encoding lentivirus where the siRNA activity may interfere with the lentiviral life cycle. In yet a further aspect, the invention provides methods for expression of a small RNA molecule within a cell, such as an siRNA capable of downregulating CCR5, wherein expression of the small RNA molecule is relatively non-cytotoxic to the cell. The invention also includes small RNA molecules, such as an siRNA capable of downregulating CCR5, that are relatively non-cytotoxic to cells.
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公开(公告)号：US20120322854A1
公开(公告)日：2012-12-20
申请号：US13526347
申请日：2012-06-18
IPC分类号： A61K31/7088 , A61P1/00 , A61P19/02 , A61P25/00
CPC分类号： A61K31/7088
摘要： The present disclosure relates to regulation of macrophage activation by delivering of miRNAs, for example miR-125b or anti-miR-125b, to macrophages. For example, in some embodiments, macrophage activation can be elevated or reduced by administering miR-125b or anti-miR-125b oligonucleotides. Also disclosed are methods for promoting T cell activation and method for treating various disorders such as tumor and autoimmune diseases.
摘要翻译： 本公开涉及通过将巨噬细胞递送miRNA（例如miR-125b或抗miR-125b）来调节巨噬细胞活化。 例如，在一些实施方案中，可通过施用miR-125b或抗miR-125b寡核苷酸来升高或降低巨噬细胞活化。 还公开了用于促进T细胞活化的方法和用于治疗各种疾病如肿瘤和自身免疫性疾病的方法。
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6.发明申请公开(公告)号：US20120065245A1
公开(公告)日：2012-03-15
申请号：US13230673
申请日：2011-09-12
申请人： David Baltimore , Ryan M. O'Connell
发明人： David Baltimore , Ryan M. O'Connell
IPC分类号： A61K31/7088 , A61P35/02 , A61P35/00 , A61P7/06 , A61P7/00
CPC分类号： A61K31/7105 , C12N2310/141
摘要： The present disclosure relates to regulation of functions of hematopoietic stem cells (HSCs) by delivering of miRNAs, including miR-125b, miR-126, and miR-155, to HSCs. For example, in some embodiments, blood output in a mammal can be increased by administering miR-125b, miR-126, and/or miR-155 oligonucleotides. Also disclosed are methods for promoting hematopoietic stem cell engraftment and method for treating a myeloproliferative disorder.
摘要翻译： 本公开涉及通过向HSC递送miRNA（包括miR-125b，miR-126和miR-155）来调节造血干细胞（HSC）的功能。 例如，在一些实施方案中，可以通过施用miR-125b，miR-126和/或miR-155寡核苷酸来增加哺乳动物的血液输出。 还公开了促进造血干细胞移植的方法和治疗骨髓增生性疾病的方法。
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公开(公告)号：US20100203630A1
公开(公告)日：2010-08-12
申请号：US12622379
申请日：2009-11-19
申请人： Xin Luo , Lili Yang , David Baltimore
发明人： Xin Luo , Lili Yang , David Baltimore
IPC分类号： C12N5/10 , C12N5/0781
CPC分类号： C12N5/0635 , C07K16/00 , C07K16/1036 , C07K16/1045 , C12N2501/056 , C12N2501/125 , C12N2501/145 , C12N2501/22 , C12N2501/23 , C12N2501/26 , C12N2501/52 , C12N2502/1394 , C12N2510/02
摘要： The present disclosure relates to systems, methods and compositions for the generation of antibody-producing B cells in vitro. Some embodiments are related to an in vitro system for generating antibody-producing B cells from hematopoietic stem/progenitor cells (HSPCs).
摘要翻译： 本公开涉及用于在体外产生产生抗体的B细胞的系统，方法和组合物。 一些实施方案涉及用于从造血干细胞/祖细胞（HSPC）产生产生抗体的B细胞的体外系统。
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公开(公告)号：US07732207B2
公开(公告)日：2010-06-08
申请号：US11689407
申请日：2007-03-21
申请人： Xiao-Feng Qin , David Baltimore , Irvin S. Y. Chen , Dong Sung An
发明人： Xiao-Feng Qin , David Baltimore , Irvin S. Y. Chen , Dong Sung An
CPC分类号： C12N15/86 , A61K38/00 , A61K48/00 , C12N7/00 , C12N15/113 , C12N15/1132 , C12N15/1138 , C12N15/867 , C12N2310/111 , C12N2310/14 , C12N2310/53 , C12N2310/531 , C12N2330/30 , C12N2740/15043 , C12N2740/16043 , C12N2830/003 , C12N2830/006 , C12N2830/008 , C12N2830/30 , C12N2830/48 , C12N2830/60 , C12N2830/85 , C12N2840/20 , C12N2840/203 , C12Q1/6897 , Y02A50/465
摘要： In one aspect, the invention provides methods and compositions for the expression of small RNA molecules within a cell using a retroviral vector (FIG. 1A). The methods can be used to express double stranded RNA complexes. Small interfering RNA (siRNA) can be expressed using the methods of the invention within a cell, that interfere with a viral life cycle by down regulating either the viral genome, a viral genome transcript, or a host cell that. In another aspect the invention provides methods for treating patients having suffering from infection, particularly infection with HIV. In a further aspect, the invention provides methods for producing siRNA encoding lentivirus where the siRNA activity may interfere with the lentiviral life cycle.
摘要翻译： 一方面，本发明提供了使用逆转录病毒载体在细胞内表达小RNA分子的方法和组合物（图1A）。 该方法可用于表达双链RNA复合物。 可以使用本发明的方法在细胞内表达小干扰RNA（siRNA），其通过下调病毒基因组，病毒基因组转录物或宿主细胞来干扰病毒生命周期。 另一方面，本发明提供了治疗患有感染，特别是HIV感染的患者的方法。 另一方面，本发明提供了用于产生编码慢病毒的siRNA的方法，其中siRNA活性可能干扰慢病毒生命周期。
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公开(公告)号：US07732193B2
公开(公告)日：2010-06-08
申请号：US10243816
申请日：2002-09-13
CPC分类号： C12N15/86 , A61K38/00 , A61K48/00 , C12N7/00 , C12N15/113 , C12N15/1132 , C12N15/1138 , C12N15/867 , C12N2310/111 , C12N2310/14 , C12N2310/53 , C12N2310/531 , C12N2330/30 , C12N2740/15043 , C12N2740/16043 , C12N2830/003 , C12N2830/006 , C12N2830/008 , C12N2830/30 , C12N2830/48 , C12N2830/60 , C12N2830/85 , C12N2840/20 , C12N2840/203 , C12Q1/6897 , Y02A50/465
摘要： The invention provides methods and compositions for the expression of small RNA molecules within a cell using a lentiviral vector. The methods can be used to express doubles stranded RNA complexes. Small interfering RNA (siRNA) can be expressed using the methods of the invention within a cell, which are capable of down regulating the expression of a target gene through RNA interference. A variety of cells can be treated according to the methods of the invention including embryos, embryogenic stem cells, allowing for the generation of transgenic animals or animals constituted partly by the transduced cells that have a specific gene or a group of genes down regulated.
摘要翻译： 本发明提供了使用慢病毒载体在细胞内表达小RNA分子的方法和组合物。 该方法可用于表达双链RNA复合物。 可以使用本发明的方法在细胞内表达小干扰RNA（siRNA），其能够通过RNA干扰下调靶基因的表达。 可以根据本发明的方法处理各种细胞，包括胚胎，胚胎干细胞，允许产生部分由转导细胞组成的转基因动物或动物，所述转导细胞具有特定基因或一组基因下调。
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公开(公告)号：US20100120122A1
公开(公告)日：2010-05-13
申请号：US12688689
申请日：2010-01-15
申请人： Pin Wang , Lili Yang , David Baltimore
发明人： Pin Wang , Lili Yang , David Baltimore
IPC分类号： C12N7/01
CPC分类号： C12N15/86 , A61K31/70 , A61K35/76 , A61K38/177 , A61K38/1774 , A61K38/178 , A61K38/1793 , A61K38/191 , A61K38/193 , A61K38/20 , A61K38/2013 , A61K38/2026 , A61K38/204 , A61K38/2046 , A61K38/2086 , A61K39/21 , A61K2039/5256 , C07K14/005 , C12N7/00 , C12N2740/13043 , C12N2740/13045 , C12N2740/15041 , C12N2740/15043 , C12N2740/15045 , C12N2770/36122 , C12N2770/36134 , C12N2810/609 , C12N2810/855 , Y02A50/386 , Y02A50/396 , Y02A50/401 , Y02A50/403 , Y02A50/407 , Y02A50/41 , Y02A50/412 , Y02A50/423 , Y02A50/466 , Y02A50/475 , Y02A50/48 , Y02A50/481 , Y02A50/492 , A61K2300/00
摘要： Methods and compositions are provided for delivery of a polynucleotide encoding a gene of interest, typically an antigen, to a dendritic cell (DC). The virus envelope comprises a DC-SIGN specific targeting molecule. The methods and related compositions can be used to treat patients suffering from a wide range of conditions, including infection, such as HIV/AIDS, and various types of cancers.
摘要翻译： 提供了将编码感兴趣的基因（通常为抗原）的多核苷酸递送至树突状细胞（DC）的方法和组合物。 病毒包膜包含DC-SIGN特异性靶向分子。 方法和相关组合物可用于治疗患有广泛病症的患者，包括感染，例如HIV / AIDS和各种类型的癌症。
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