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    Regulation of hematopoietic stem cell functions through microRNAs
    63.
    发明授权
    Regulation of hematopoietic stem cell functions through microRNAs 有权
    通过microRNA调节造血干细胞功能

    公开(公告)号：US09006195B2

    公开(公告)日：2015-04-14

    申请号：US13230673

    申请日：2011-09-12

    Applicant: David Baltimore ,  Ryan M. O'Connell

    Inventor： David Baltimore ,  Ryan M. O'Connell

    IPC: A61K31/70 ,  A61K31/7105

    CPC classification number: A61K31/7105 ,  C12N2310/141

    Abstract: 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.

    Abstract translation: 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。

    Method of targeted gene delivery using viral vectors
    64.
    发明授权
    Method of targeted gene delivery using viral vectors 有权
    使用病毒载体的靶向基因递送方法

    公开(公告)号：US08821856B2

    公开(公告)日：2014-09-02

    申请号：US13041115

    申请日：2011-03-04

    Applicant: David Baltimore ,  Pin Wang ,  Lili Yang

    Inventor： David Baltimore ,  Pin Wang ,  Lili Yang

    IPC: C12N15/867 ,  C07K16/28 ,  C07K17/02 ,  A61K35/76 ,  C07K14/155

    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。

    System and method for analyzing a biological sample
    67.
    发明授权
    System and method for analyzing a biological sample 有权
    用于分析生物样品的系统和方法

    公开(公告)号：US08313643B1

    公开(公告)日：2012-11-20

    申请号：US12589234

    申请日：2009-10-20

    Applicant: David Baltimore

    Inventor： David Baltimore

    IPC: G01N37/00

    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。

    USE OF SAM68 FOR MODULATING SIGNALING THROUGH THE TNF RECEPTOR
    69.
    发明申请
    USE OF SAM68 FOR MODULATING SIGNALING THROUGH THE TNF RECEPTOR 有权
    使用SAM68通过TNF受体进行信号调节

    公开(公告)号：US20120121608A1

    公开(公告)日：2012-05-17

    申请号：US13284792

    申请日：2011-10-28

    Applicant: Parameswaran Ramakrishnan ,  David Baltimore

    Inventor： Parameswaran Ramakrishnan ,  David Baltimore

    IPC: A61K39/395 ,  A61K31/7088 ,  A61P35/00 ,  G01N33/566 ,  C12Q1/68 ,  G01N33/573 ,  C12Q1/06 ,  A61K31/713 ,  A61K31/7105

    CPC classification number: A61K31/7105 ,  C12N15/1135 ,  C12N2310/14

    Abstract: Sam68 plays a role in TNF-dependent signaling, including NF-kB signaling and extrinsic activation of apoptosis. In some embodiments, inhibitors of Sam68 are administered to inhibit TNF-dependent signaling, for example to inhibit NF-kB signaling or apoptosis in a patient in need. In some embodiments, functional Sam68 is administered to increase TNF-dependent signaling, for example to induce apoptosis in a patient in need. In some embodiments, methods are provided determining whether the TNF-dependent or TNF-independent branch of a signaling pathway is active in a cell or cells, or for drug screening applications.

    Abstract translation: Sam68在TNF依赖性信号传导中起重要作用，包括NF-kB信号传导和凋亡的外在激活。 在一些实施方案中，施用Sam68的抑制剂以抑制TNF依赖性信号传导，例如抑制需要的患者中的NF-kB信号传导或凋亡。 在一些实施方案中，施用功能性Sam68以增加TNF依赖性信号传导，例如在需要的患者中诱导细胞凋亡。 在一些实施方案中，提供了确定信号传导途径的TNF依赖性或不依赖于TNF的分支在细胞或细胞中活性或用于药物筛选应用的方法。

    TREATMENT OF AUTOIMMUNE INFLAMMATION USING MIR-155
    70.
    发明申请
    TREATMENT OF AUTOIMMUNE INFLAMMATION USING MIR-155 审中-公开
    使用MIR-155治疗自身免疫炎症

    公开(公告)号：US20120064122A1

    公开(公告)日：2012-03-15

    申请号：US13230695

    申请日：2011-09-12

    Applicant: David Baltimore ,  Ryan M. O'Connell ,  Daniel Kahn

    Inventor： David Baltimore ,  Ryan M. O'Connell ,  Daniel Kahn

    IPC: A61K31/7088 ,  A61P37/04 ,  A61P31/00 ,  A61P31/04 ,  A61P31/12 ,  C12N5/0784 ,  A61P25/00 ,  A61P19/02 ,  A61P1/00 ,  A61P17/06 ,  C12N5/0783 ,  A61P29/00 ,  A61P37/00

    CPC classification number: A61K31/7088 ,  C12N15/113 ,  C12N2310/113

    Abstract: The present disclosure relates to the finding that microRNA-155 plays a role in the development and activity of CD4+ T cells. CD4+ T cell development and function, particularly TH17 and TH1 T cell development, can be modulated by delivery of microRNA-155 (miR-155) or antisense miR-155 to target CD4+ cells or precursor cells. In some embodiments, antisense miR-155 is used to reduce tissue specific autoimmune inflalmmation and to treat autoimmune disease. In addition, miR155 and antisense miR-155 can be used to modulate expression of cytokines from dendritic cells.

    Abstract translation: 本公开涉及microRNA-155在CD4 + T细胞的发育和活性中起作用的发现。 可通过将microRNA-155（miR-155）或反义miR-155递送至靶向CD4 +细胞或前体细胞来调节CD4 + T细胞发育和功能，特别是TH17和TH1T细胞发育。 在一些实施方案中，反义miR-155用于减少组织特异性自身免疫炎症并治疗自身免疫性疾病。 此外，miR155和反义miR-155可用于调节来自树突状细胞的细胞因子的表达。

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