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    PRODUCTS FOR THE DELIVERY OF THERAPEUTIC/DIAGNOSTIC COMPOUNDS TO THE HEART
    31.
    发明申请
    PRODUCTS FOR THE DELIVERY OF THERAPEUTIC/DIAGNOSTIC COMPOUNDS TO THE HEART 审中-公开
    用于向心脏递送治疗/诊断化合物的产品

    公开(公告)号：WO2016102576A1

    公开(公告)日：2016-06-30

    申请号：PCT/EP2015/080991

    申请日：2015-12-22

    申请人： CONSIGLIO NAZIONALE DELLE RICERCHE ,  ISTITUTO NAZIONALE ASSICURAZIONE CONTRO GLI INFORTUNI SUL LAVORO

    发明人： CATALUCCI, Daniele ,  MIRAGOLI, Michele ,  IAFISCO, Michele ,  TAMPIERI, Anna

    IPC分类号： A61K9/14 ,  A61K9/51

    CPC分类号： A61K9/5115 ,  A61K9/007 ,  A61K9/143 ,  A61K9/5192 ,  A61K31/7105 ,  A61K38/03 ,  A61K47/52 ,  A61K47/6929 ,  C12N15/113 ,  C12N2310/141 ,  C12N2320/32

    摘要： The invention relates to a process for the preparation of a product comprising one or more nanoparticles of calcium phosphate (CaP-NP) with negative surface charge having a ζ-potential in the range from -41.0 mV to -27.0 mV comprising the steps of: a) maintaining a mixture having a pH in the range from 7 to 10 and comprising an aqueous solution of calcium, an aqueous solution of phosphate and a solution of citrate ions at a temperature in the range from 20 °C to 40 °C for a time in the range from 30 seconds to 10 minutes; b) removing non-reacted ions from the solution of step a), thus obtaining a suspension of one or more nanoparticles of calcium phosphate (CaP-NP); c) recovering the product of one or more nanoparticles of calcium phosphate (CaP-NP) from the suspension of step b). In an advantageous embodiment, the process of the invention provides, in the mixture of step a), also an aqueous solution of one or more therapeutic/diagnostic compounds. The product of the invention may be used as a vehicle for one or more diagnostic/therapeutic compounds for the treatment of cardiovascular diseases through inhalation administration.

    摘要翻译： 本发明涉及一种制备包含一种或多种具有负电荷的磷酸钙（CaP-NP）的纳米颗粒的产品的方法，所述磷酸钙（CaP-NP）具有-41.0mV至-27.0mV范围内的ζ电位，包括以下步骤： a）保持pH在7至10范围内的混合物，并在20℃至40℃的温度范围内包含钙水溶液，磷酸盐水溶液和柠檬酸根离子溶液，用于 时间范围从30秒到10分钟; b）从步骤a）的溶液中除去未反应的离子，从而获得一种或多种磷酸钙（CaP-NP）纳米颗粒的悬浮液; c）从步骤b）的悬浮液中回收一种或多种磷酸钙（CaP-NP）纳米颗粒的产物。 在有利的实施方案中，本发明的方法在步骤a）的混合物中还提供一种或多种治疗/诊断化合物的水溶液。 本发明的产品可以用作一种或多种用于通过吸入给药治疗心血管疾病的诊断/治疗化合物的载体。

    LIGAND-MODIFIED DOUBLE-STRANDED NUCLEIC ACIDS
    32.
    发明申请
    LIGAND-MODIFIED DOUBLE-STRANDED NUCLEIC ACIDS 审中-公开
    配体改性的双链核酸

    公开(公告)号：WO2016100401A1

    公开(公告)日：2016-06-23

    申请号：PCT/US2015/065906

    申请日：2015-12-15

    申请人： DICERNA PHARMACEUTICALS, INC.

    发明人： BROWN, Bob, Dale ,  WANG, Weimin

    IPC分类号： C12N15/11 ,  C12N15/113

    CPC分类号： C07H21/04 ,  C07H1/00 ,  C07H99/00 ,  C12N15/111 ,  C12N15/113 ,  C12N2310/14 ,  C12N2310/351 ,  C12N2310/3515 ,  C12N2310/53 ,  C12N2310/533 ,  C12N2320/32 ,  C12N2320/51 ,  C12N2330/30

    摘要： The invention provides for double stranded nucleic acid molecules comprising a 5 'extension of the sense or antisense strand and further comprising a plurality of nucleotides that are conjugated to a ligand and methods of using the double-stranded nucleic acid molecules. Ligand-modified oligomers where the sense stands form a tetraloop provide new potent and stable RNA interference agents. These dsNA molecules are synthesized using a plurality of nucleotides that include ligand-modified monomers, nucleotide analog monomers, modified nucleotide monomers and the like, using standard nucleotide synthetic methods and systems.

    摘要翻译： 本发明提供了包含正义或反义链的5'延伸的双链核酸分子，并且还包含与配体缀合的多个核苷酸和使用双链核酸分子的方法。 配体修饰的寡聚物，其中有义位形成四氢叶酸提供新的有力和稳定的RNA干扰剂。 使用标准核苷酸合成方法和系统，使用包括配体改性单体，核苷酸类似单体，修饰的核苷酸单体等的多个核苷酸合成这些dsNA分子。

    USE AND PRODUCTION OF CHD8+/- TRANSGENIC ANIMALS WITH BEHAVIORAL PHENOTYPES CHARACTERISTIC OF AUTISM SPECTRUM DISORDER
    39.
    发明申请
    USE AND PRODUCTION OF CHD8+/- TRANSGENIC ANIMALS WITH BEHAVIORAL PHENOTYPES CHARACTERISTIC OF AUTISM SPECTRUM DISORDER 审中-公开
    自发性谱系疾病行为表型特征的CHD8 +/-转基因动物的使用和生产

    公开(公告)号：WO2016049163A2

    公开(公告)日：2016-03-31

    申请号：PCT/US2015051691

    申请日：2015-09-23

    申请人： BROAD INST INC ,  MASSACHUSETTS INST TECHNOLOGY

    发明人： PLATT RANDALL JEFFREY ,  ZHANG FENG ,  SLAYMAKER IAN

    IPC分类号： C12N15/10

    CPC分类号： C12N15/63 ,  A01K67/0275 ,  A01K2217/072 ,  A01K2227/105 ,  A01K2267/0306 ,  A01K2267/0318 ,  C12N9/22 ,  C12N15/111 ,  C12N2310/10 ,  C12N2310/20 ,  C12N2320/11 ,  C12N2320/32 ,  C12N2330/51 ,  C12N2740/15041 ,  C12N2750/14341

    摘要： The invention involves inducing a plurality e.g., 3-50 or more mutations (e.g., any whole number between 3 and 50 or more of mutations, with it noted that in some embodiments there can be up to 16 different RNA(s), e.g., sgRNAs each having its own a promoter, in a vector, such an AAV vector or a lentiviral vector and that when each sgRNA does not have its own promoter, there can be twice to thrice that amount of different RNA(s), e.g., sgRNAs, e.g., 32 or even 48 different guides delivered by one vector) in transgenic Cas9 eukaryotes to model a neuronal disease or disorder. The invention comprehends testing putative treatments with such models, e.g., testing putative chemical compounds that may be pharmaceutically relevant for treatment or gene therapy that may be relevant for treatment, or combinations thereof. The invention allows for the study of genetic diseases and putative treatments to better understand and alleviate a genetic disease or a condition, e.g., autism, autism-spectrum disease or disorder, obsessive compulsive disorder, or psychiatric disorders.

    摘要翻译： 本发明涉及诱导多个例如3-50个或更多个突变（例如，3至50个或更多个突变之间的任何整数），其中注意到在一些实施方案中可以存在多达16个不同的RNA，例如， 在载体，如AAV载体或慢病毒载体中各自具有其自身启动子的sgRNA，并且当每个sgRNA不具有其自身启动子时，可存在两倍至三倍的不同RNA，例如sgRNA ，例如，由一个载体递送的32个或甚至48个不同的指导）转染到转基因Cas9真核生物中以模拟神经元疾病或病症。 本发明包括用这些模型测试假定的治疗，例如测试可能与治疗或可能与治疗相关的基因治疗在药学上相关的推定化学化合物或其组合。 本发明允许研究遗传疾病和推定的治疗以更好地理解和缓解遗传疾病或病症，例如孤独症，自闭症谱系疾病或障碍，强迫症或精神障碍。