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    TWO-DIMENSIONAL POLYMERIC STRUCTURES AND METHOD FOR PRODUCING THEREOF
    96.
    发明申请
    TWO-DIMENSIONAL POLYMERIC STRUCTURES AND METHOD FOR PRODUCING THEREOF 审中-公开
    二维聚合物结构及其生产方法

    公开(公告)号：WO2016042484A1

    公开(公告)日：2016-03-24

    申请号：PCT/IB2015/057096

    申请日：2015-09-15

    申请人： ECOLE POLYTECHNIQUE FEDERALE DE LAUSANNE (EPFL)

    发明人： PU, Ye ,  ZIELINSKI, Marcin Stefan ,  PSALTIS, Demetri ,  HUBBELL, Jeffrey Alan

    IPC分类号： A61K9/50 ,  A61K9/51

    CPC分类号： A61K9/5192 ,  A61K9/501 ,  A61K9/5115 ,  Y02A50/385 ,  Y02A50/387 ,  Y02A50/465 ,  Y02A50/471 ,  Y02A50/481

    摘要： The present invention relates to polymeric structures, in the form of flat membrane-like surfaces or micro- nanostructures such as capsules, characterized in that it comprises a substantially two-dimensional layer of covalently-bonded monomers of R-substituted metal or metalloid oxides. Said polymeric structures in most embodiments have a crystal architecture with a hexagonal lattice, but the nature of the covalent bonds present therein impart a bending flexibility that make the polymeric structures behave as a "soft" crystal. Methods of producing such structures, composition comprising thereof and method of using thereof are also included within the present disclosure.

    摘要翻译： 本发明涉及以扁平膜状表面或微纳米结构如胶囊形式的聚合物结构，其特征在于其包含R取代金属或准金属氧化物共价键合的单体的基本二维层。 所述聚合物结构在大多数实施方案中具有六方晶格的晶体结构，但是其中存在的共价键的性质赋予了使聚合物结构表现为“软”晶体的弯曲柔性。 生产此类结构的方法，包含其的组合物及其使用方法也包括在本公开内。

    METHODS OF LIMITING MORBIDITY IN HEMOGLOBINOPATHIES
    97.
    发明申请
    METHODS OF LIMITING MORBIDITY IN HEMOGLOBINOPATHIES 审中-公开
    限制HEMOGLOBINOPATHIES中的MORBIDITY的方法

    公开(公告)号：WO2015179855A1

    公开(公告)日：2015-11-26

    申请号：PCT/US2015/032366

    申请日：2015-05-26

    申请人： DUKE UNIVERSITY

    发明人： ZENNADI, Rahima

    IPC分类号： A61P7/06 ,  A61K31/352 ,  A61K31/4412 ,  A61K31/166 ,  A61K31/404

    CPC分类号： A61K31/18 ,  A61K31/137 ,  A61K31/138 ,  A61K31/17 ,  A61K31/277 ,  A61K31/404 ,  A61K31/44 ,  A61K31/60 ,  A61K31/713 ,  A61K45/06 ,  G01N33/573 ,  G01N2800/52 ,  Y02A50/471 ,  A61K2300/00

    摘要： Methods of alleviating the symptoms of hemoglobinopathies, including, but not limited to, sickle cell disease, β-thalassemia, and hemoglobin H disease are provided, In some embodiments, the methods comprise administering an agent to the subject if the subject has increased expression or activation of at least one of ERK, Ras, BRAF, Rail, MEK, β-arrestin 1/2, Syk, Ρ60-c-Src, or GRK2. Methods of determining the likelihood of a complication or vascular endothelial injury and mortality resulting from a hemoglobinopathy in a subject are also provided.

    摘要翻译： 提供了减轻血红蛋白病症状的方法，包括但不限于镰状细胞病，β-地中海贫血和血红蛋白H疾病。在一些实施方案中，所述方法包括如果受试者具有增加的表达或 激活ERK，Ras，BRAF，Rail，MEK，β-arrestin 1/2，Syk，P60-c-Src或GRK2中的至少一种。 还提供了确定并发症或血管内皮损伤的可能性以及由受试者中的血红蛋白病引起的死亡的方法。

    COMPOSITIONS AND METHODS FOR SUPPRESSION OF INHIBITOR FORMATION AGAINST FACTOR VIII IN HEMOPHILIA A PATIENTS
    99.
    发明申请
    COMPOSITIONS AND METHODS FOR SUPPRESSION OF INHIBITOR FORMATION AGAINST FACTOR VIII IN HEMOPHILIA A PATIENTS 审中-公开
    抑制HEMOPHILIA A患者抑制因子VIII的抑制剂形成的组合物和方法

    公开(公告)号：WO2015073988A1

    公开(公告)日：2015-05-21

    申请号：PCT/US2014/065994

    申请日：2014-11-17

    申请人： TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA ,  OFFICE OF TECHNOLOGY LICENSING ,  HERZOG, Roland, W. ,  DANIELL, Henry

    发明人： HERZOG, Roland, W. ,  DANIELL, Henry

    IPC分类号： C12N5/02 ,  C12N5/00 ,  A01H9/00

    CPC分类号： C07K14/28 ,  A61K36/00 ,  A61K38/36 ,  A61K38/37 ,  A61K38/45 ,  A61K38/47 ,  C07K14/755 ,  C07K2319/00 ,  C12N15/8214 ,  C12N15/8257 ,  C12Y204/00 ,  C12Y302/0102 ,  C12Y302/01022 ,  Y02A50/471

    摘要： Protein replacement therapy for patients with hemophilia or other inherited protein deficiencies is often complicated by pathogenic antibody responses, including antibodies that neutralize the therapeutic protein or that predispose to potentially life -threatening anaphylactic reactions by formation of lgE. Using murine hemophilia as a model, we have developed a prophylactic protocol against such responses that is non-invasive and does not include immune suppression or genetic manipulation of the patient's cells. Oral delivery of coagulation factor expressed in chloroplasts, bioencapsulated in plant cells, effectively blocked formation of inhibitory antibodies in protein replacement therapy. Inhibitor titers were mostly undetectable and up to 100-fold lower in treated subjects when compared to controls. Moreover, this treatment eliminated fatal anaphylactic reactions that occurred after 4 to 6 exposures to intravenous coagulation factor protein.

    摘要翻译： 血友病或其他遗传性蛋白质缺乏症患者的蛋白质替代治疗通常由致病性抗体反应复杂化，包括中和治疗性蛋白质的抗体或易于通过形成lgE而潜在的生命危险的过敏反应。 使用鼠血友病作为模型，我们制定了一种针对非侵入性反应的预防方案，不包括对患者细胞的免疫抑制或遗传操作。 在植物细胞中生物封装的叶绿体中表达的凝血因子的口服递送在蛋白质替代疗法中有效阻断了抑制性抗体的形成。 与对照相比，抑制剂滴度几乎不可检测，并且在治疗的受试者中高达100倍。 此外，这种治疗消除了4至6次静脉内凝血因子蛋白暴露后发生的致命过敏反应。