公开(公告)号：US20100267146A1

公开(公告)日：2010-10-21

申请号：US12769157

申请日：2010-04-28

Applicant: Carlos Lois-Caballe ,  David Baltimore ,  Xiao-Feng Qin ,  Irvin S.Y. Chen ,  Dong Sung An

Inventor： Carlos Lois-Caballe ,  David Baltimore ,  Xiao-Feng Qin ,  Irvin S.Y. Chen ,  Dong Sung An

IPC: C12N15/63

CPC classification number: 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

Abstract: 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.

Abstract translation: 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）。 可以使用本发明的方法在细胞内表达小干扰RNA（siRNA）。 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。 在另一方面，本发明提供了在细胞内表达小RNA分子的方法，例如能够下调CCR5的siRNA，其中小RNA分子的表达对细胞是相对非细胞毒性的。 本发明还包括对细胞相对非细胞毒性的小RNA分子，例如能够下调CCR5的siRNA。

公开(公告)号：US20100120140A1

公开(公告)日：2010-05-13

申请号：US12688779

申请日：2010-01-15

Applicant: Pin Wang ,  Lili Yang ,  David Baltimore

Inventor： Pin Wang ,  Lili Yang ,  David Baltimore

IPC: C12N15/63

CPC classification number: 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

Abstract: 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.

公开(公告)号：US20080003658A1

公开(公告)日：2008-01-03

申请号：US11689407

申请日：2007-03-21

Applicant: Xiao-Feng Qin ,  David Baltimore ,  Irvin Chen ,  Dong An

Inventor： Xiao-Feng Qin ,  David Baltimore ,  Irvin Chen ,  Dong An

IPC: C12N7/00 ,  C12N5/06

CPC classification number: 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

Abstract: 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.

Abstract translation: 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。

公开(公告)号：US20070020238A1

公开(公告)日：2007-01-25

申请号：US11446353

申请日：2006-06-01

Applicant: David Baltimore ,  Ping Wang ,  Lili Yang

Inventor： David Baltimore ,  Ping Wang ,  Lili Yang

IPC: A61K48/00 ,  C12N15/867 ,  C12Q1/70 ,  C12N7/00

CPC classification number: C12N15/86 ,  A61K2039/505 ,  C07K16/2887 ,  C07K2317/53 ,  C07K2317/622 ,  C07K2319/00 ,  C07K2319/03 ,  C12N2740/15043 ,  C12N2740/16043 ,  C12N2740/16045 ,  C12N2799/027 ,  C12N2810/80 ,  C12N2810/851 ,  C12N2810/859 ,  Y02A50/386 ,  Y02A50/396

Abstract: Methods and compositions are provided for delivering a polynucleotide encoding a gene of interest to a target cell using a virus. The virus envelope comprises a cell-specific binding determinant that recognizes and binds to a component on the target cell surface, leading to endocytosis of the virus. A separate fusogenic molecule is also present on the envelope and facilitates delivery of the polynucleotide across the membrane and into the cytosol of the target cell. The methods and related compositions can be used for treating patients having suffering from a wide range of conditions, including infection, such as HIV; cancers, such as non-Hodgkin's lymphoma and breast cancer; and hematological disorders, such as severe combined immunodeficiency.

Abstract translation: 提供了使用病毒将编码目标基因的多核苷酸递送至靶细胞的方法和组合物。 病毒包膜包含识别和结合靶细胞表面上的组分的细胞特异性结合决定簇，导致病毒的内吞。 单独的融合分子也存在于包膜上并促进多核苷酸穿过膜并进入靶细胞的胞质溶胶。 方法和相关组合物可用于治疗患有广泛病症（包括HIV感染）的患者; 癌症，如非霍奇金淋巴瘤和乳腺癌; 和血液学疾病，如严重的联合免疫缺陷。

公开(公告)号：US06916917B1

公开(公告)日：2005-07-12

申请号：US09441926

申请日：1999-11-17

Applicant: David Baltimore ,  Xiaolu Yang ,  Howard Y. Chang

Inventor： David Baltimore ,  Xiaolu Yang ,  Howard Y. Chang

IPC: A61K38/48 ,  A61K39/395 ,  C07H21/04 ,  C12N9/64 ,  C12P21/06 ,  C12Q1/68

CPC classification number: C12N9/6475 ,  C07H21/04 ,  C07K2319/00 ,  C07K2319/50

Abstract: The present invention relates to a chimeric pro-caspase, which contains a pro-caspase domain and an oligomerizing domain. The invention also relates to an antibody that reacts specifically with a chimeric pro-caspase. In addition, the invention further relates to a polynucleotide encoding a chimeric pro-caspase, and to nucleotide sequences, which can hybridize specifically with a polynucleotide encoding a chimeric pro-caspase. The present invention also relates to a method of inducing apoptosis in a cell by providing a chimeric pro-caspase in the cell, wherein the chimeric pro-caspase includes a pro-caspase domain and an oligomerizing domain, whereby the chimeric pro-caspase forms an oligomer in the cell, thereby activating caspase activity of the chimeric pro-caspase and inducing apoptosis in the cell. The present invention further relates to a method of reducing the severity of a pathologic condition in a subject, by providing cells of the subject that are involved in the pathologic condition with a chimeric pro-caspase comprising a pro-caspase domain and an oligomerizing domain, whereby the chimeric pro-caspase forms an oligomer in the cells, thereby activating caspase activity of the chimeric pro-caspase, inducing apoptosis in the cells, and reducing the severity of the pathologic condition in the subject.

Abstract translation: 本发明涉及含有前半胱天冬酶蛋白酶结构域和寡聚化结构域的嵌合前半胱天冬酶。 本发明还涉及与嵌合亲胱天蛋白酶特异性反应的抗体。 此外，本发明还涉及编码嵌合型前半胱氨酸蛋白酶的多核苷酸以及可与编码嵌合型前半胱天冬酶的多核苷酸特异性杂交的核苷酸序列。 本发明还涉及通过在细胞中提供嵌合型前半胱氨酸蛋白酶诱导细胞凋亡的方法，其中所述嵌合前半胱氨酸蛋白酶包括前半胱天冬酶蛋白酶结构域和寡聚结构域，由此所述嵌合型前半胱天冬酶形成 从而激活嵌合前半胱氨酸蛋白酶的半胱天冬酶活性并诱导细胞凋亡。 本发明还涉及通过用包含前半胱天冬酶蛋白酶结构域和寡聚结构域的嵌合前半胱氨酸蛋白酶提供参与病理状况的受试者的细胞来降低受试者病理状况的严重性的方法， 由此嵌合的前半胱天冬酶在细胞中形成寡聚体，从而激活嵌合前半胱天冬酶的半胱天冬酶活性，诱导细胞凋亡，并降低受试者病理状况的严重性。

公开(公告)号：US5804374A

公开(公告)日：1998-09-08

申请号：US418266

申请日：1995-04-06

Applicant: David Baltimore ,  Ranjan Sen ,  Phillip A. Sharp ,  Harinder Singh ,  Louis Staudt ,  Jonathan H. LeBowitz ,  Albert S. Baldwin, Jr. ,  Roger G. Clerc ,  Lynn M. Corcoran ,  Patrick A. Baeuerle ,  Michael J. Lenardo ,  Chen-Ming Fan ,  Thomas P. Maniatis

Inventor： David Baltimore ,  Ranjan Sen ,  Phillip A. Sharp ,  Harinder Singh ,  Louis Staudt ,  Jonathan H. LeBowitz ,  Albert S. Baldwin, Jr. ,  Roger G. Clerc ,  Lynn M. Corcoran ,  Patrick A. Baeuerle ,  Michael J. Lenardo ,  Chen-Ming Fan ,  Thomas P. Maniatis

IPC: C12Q1/68 ,  G01N33/68 ,  C12N15/11

CPC classification number: C12Q1/6897 ,  G01N33/6872 ,  G01N2500/02

Abstract: Constitutive and tissue-specific protein factors which bind to transcriptional regulatory elements of Ig genes (promoter and enhancer) are described. The factors were identified and isolated by an improved assay for protein-DNA binding. Genes encoding factors which positively regulate transcription can be isolated and employed to enhance transription of Ig genes. In particular, NF-kB, the gene encoding NF-kB, IkB and the gene encoding IkB and uses therefor.

Abstract translation: 描述了结合Ig基因转录调控元件（启动子和增强子）的组成型和组织特异性蛋白因子。 通过改进的蛋白质-DNA结合测定鉴定和分离因子。 可以分离用于正调节转录的编码因子的基因以增强Ig基因的转录。 特别地，NF-kB，编码NF-kB，IkB的基因和编码IkB的基因并用于其。

公开(公告)号：US5230888A

公开(公告)日：1993-07-27

申请号：US530468

申请日：1990-05-29

Applicant: David Baltimore ,  Marie B. Chow

Inventor： David Baltimore ,  Marie B. Chow

IPC: A61K39/00 ,  A61K39/42 ,  C07K14/105 ,  C07K16/10

CPC classification number: C07K14/005 ,  A61K39/42 ,  C07K16/1009 ,  A61K39/00 ,  C12N2770/32622

Abstract: The production of neutralizing antibodies for poliovirus, or other enteroviruses, are disclosed herein employing the capsid polypeptide VP1. Vaccines can be produced employing this polypeptide in place of killed or live attenuated virus, and the VP1 polypeptide can be employed in assays in place of whole virions. VP1 polypeptide can be produced by isolating it from live virion or can be produced by cloning cDNA sequences coding for this protein in recombinant cDNA vectors.

Abstract translation: 本文公开了使用衣壳多肽VP1产生脊髓灰质炎病毒或其他肠道病毒的中和抗体。 可以使用该多肽代替杀死的或活的减毒病毒产生疫苗，并且VP1多肽可以用于测定而不是整个病毒体。 VP1多肽可以通过从活病毒体分离而产生，或者可以通过在重组cDNA载体中克隆编码该蛋白的cDNA序列来产生。

公开(公告)号：US08865881B2

公开(公告)日：2014-10-21

申请号：US13400945

申请日：2012-02-21

Applicant: Alejandro Benjamin Balazs ,  David Baltimore

Inventor： Alejandro Benjamin Balazs ,  David Baltimore

IPC: C07H21/04 ,  C12N15/63 ,  C07K16/10 ,  A61K48/00

CPC classification number: C07K16/109 ,  C07K16/1045 ,  C12N7/00 ,  C12N2750/14141 ,  C12N2750/14143

Abstract: Disclosed herein are compositions, systems and methods for delivery of proteins of interest using adeno-associated virus (AAV) vectors.

Abstract translation: 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。

公开(公告)号：US20130316366A1

公开(公告)日：2013-11-28

申请号：US13901940

申请日：2013-05-24

Applicant: Kenneth Yu ,  David Baltimore ,  Lili Yang

Inventor： Kenneth Yu ,  David Baltimore ,  Lili Yang

IPC: C12N15/85 ,  G01N33/68 ,  G01N33/58 ,  C12N15/86 ,  C12N15/90

CPC classification number: C12N15/85 ,  C07K2319/00 ,  C12N15/86 ,  C12N15/907 ,  C12N2740/15043 ,  G01N33/582 ,  G01N33/68

Abstract: 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.

Abstract translation: 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。

公开(公告)号：US20130065316A1

公开(公告)日：2013-03-14

申请号：US13673056

申请日：2012-11-09

Applicant: David Baltimore

Inventor： David Baltimore

IPC: G01N35/00

CPC classification number: G01N33/487 ,  Y10T436/11

Abstract: 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.

Abstract translation: 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。