专利号:US-2022160788-A1 优先权日:2019-03-22 标题 :Bifunctional vectors allowing bcl11a silencing and expression of an anti-sickling hbb and uses thereof for gene therapy of b-hemoglobinopathies 发明人:MICCIO ANNARITA; AMENDOLA MARIO; BRUSSON MÉGANE; CAVAZZANA MARINA; MAVILIO FULVIO 权利人:INST NAT SANTE RECH MED; UNIV DEVRY VAL DESSONNE; ASSIST PUBLIQUE HOPITAUX PARIS APHP; UNIV PARIS; FOND IMAGINE 摘要:The #β-hemoglobinopathies #β-thalassemia (BT) and sickle cell disease (SCD) are the most frequent genetic disorders worldwide. These diseases are caused by mutations causing reduced or abnormal synthesis of the β-globin chain of the adult hemoglobin (Hb) tetramer. Here, the inventors intend to improve HSC-based gene therapy for β-thalassemia and SCD by developing an innovative, highly infectious LV vector expressing a potent anti-sickling β-globin transgene and a second biological function either increasing fetal γ-globin expression (for β-thalassemia and SCD). More particularly, the inventors have designed a novel lentivirus (LV), which carry two different functions: βAS3 gene addition and gene silencing. This last strategy allows the re-expression of the fetal γ-globin genes (HBG1 and HBG2) and production of the endogenous fetal hemoglobin (HbF). Elevated levels of HbF and HbAS3 (Hb tetramer containing βAS3-globin) will benefit the β-hemoglobinopathy phenotype by increasing the total amount of β-like globin that will: (i) reduce the alpha precipitates and improve the alpha/non alpha ratio in β-thalassemia, and (ii) reduce the sickling in SCD. This combined strategy will improve the β-hemoglobinopathy phenotype at a lower vector copy number (VCN) per cell compared to a LV expressing the βAS3 alone.
专利号:US-2023271983-A1 优先权日:2020-07-29 标题 :Functionalized isonitriles and products, preparation and uses thereof 发明人:SOTELO PÉREZ EDDY; AZUAJE GUERRERO JHONNY ALBERTO; MAJELLARO MARIA 权利人:UNIV SANTIAGO COMPOSTELA 摘要:The present invention relates to functionalized isonitrile compounds, formed by means of multicomponent environmentally friendly reactions, suitable for coupling to functional molecules such as biomolecules, APIs, chromophore and fluorophore molecules. The invention also relates to conjugates between said isonitrile compounds and functional molecules, which are useful in the synthesis of pharmaceutic drugs or tools, fluorescent molecules and labels, polymeric smart materials. The invention furthermore provides a kit for the in-vitro preparation of the functionalized isontrile compounds and conjugates. The invention also contemplates the medical use of said compounds and conjugates.
专利号:US-9907753-B2 优先权日:2010-03-19 标 题 :Lipid vesicle compositions and methods of use 发明人:IRVINE DARRELL J; MOON JAEHYUN 权利人:MASSACHUSETTS INST TECHNOLOGY 摘要:The invention provides delivery systems comprised of stabilized multilamellar vesicles, as well as compositions, methods of synthesis, and methods of use thereof. The stabilized multilamellar vesicles may comprise prophylactic, therapeutic and/or diagnostic agents.
专利号:US-7993390-B2 优先权日:2002-02-08 标 题:Implantable or insertable medical device resistant to microbial growth and biofilm formation 发明人:MILLER KATHLEEN M; BUCAY-COUTO WEENNA; LI JIANMIN 权利人:BOSTON SCIENT SCIMED INC 摘要:Disclosed are implantable or insertable medical devices that provide resistance to microbial growth on and in the environment of the device and resistance to microbial adhesion and biofilm formation on the device. In particular, the invention discloses implantable or insertable medical devices that comprise at least one biocompatible matrix polymer region, an antimicrobial agent for providing resistance to microbial growth and/or a microbial adhesion/biofilm synthesis inhibitor for inhibiting the attachment of microbes and the synthesis and accumulation of biofilm on the surface of the medical device. Also disclosed are methods of manufacturing such devices under conditions that substantially prevent preferential partitioning of any of said bioactive agents to a surface of the biocompatible matrix polymer and substantially prevent chemical modification of said bioactive agents.
专利号:US-6277489-B1 优先权日:1998-12-04 标题:Support for high performance affinity chromatography and other uses 发明人:ABBOTT NICHOLAS; STROEVE PIETER; DUBROVSKY TIMOTHY B; HOU ZHIZHONG 权利人:UNIV CALIFORNIA 摘要:Multilayered particulate materials are formed by coating a particulate substrate with a metal and adsorbing an organic layer comprising a recognition moiety onto the metal film. The recognition moiety interacts with an analyte of interest allowing for its detection, purification, etc. Suitable recognition moieties can be selected from a range of species including, small molecules, polymers and biomolecules and the like. The novel particulate materials of the invention can be utilized in an array of methods including, ion-exchange, ion-selective ion-exchange, assays, affinity dialysis, size exclusion dialysis, as supports in solid phase synthesis, combinatorial synthesis and screening of compound libraries and the like.
专利号:US-2011263650-A1 优先权日:2007-07-20 标 题:Tubulysin D Analogues 发明人:ELLMAN JONATHAN A; PATTERSON ANDREW W; PELTIER HILLARY; SASSE FLORENZ 权利人:HELMHOLTZ INFEKTIONSFORSCHUNG 摘要:The present invention provides novel tubulysin analogues, methods of making and methods of using such analogues and conjugates thereof. The essential features for the potent cytotoxicity of tubulysin D have been established for the first time by the synthesis and evaluation of a series of analogues. By identifying functionality that surprisingly is not necessary for activity, highly potent cell-growth inhibitors have been developed that are smaller and considerably more stable than tubulysin D.
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合成参考文献
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