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    • 61. 发明申请
    • SPLICEOSOME MEDIATED RNA TRANS-SPLICING IN STEM CELLS
    • 干细胞中SPLICEOSOME介导的RNA转移分离
    • WO2003104416A2
    • 2003-12-18
    • PCT/US2003/017954
    • 2003-06-05
    • INTRONN, INC.MITCHELL, Lloyd, G.ENGLEHARDT, JohnLIU, Xiao, Ming
    • MITCHELL, Lloyd, G.ENGLEHARDT, JohnLIU, Xiao, Ming
    • C12N
    • C12N5/0688A61K48/005C12N2510/02
    • The present invention provides methods and compositions for generating novel nucleic acid molecules through targeted spliceosomal mediated trans -splicing in stem cells. The compositions of the invention include stem cells engineered to express pre- trans -splicing molecules (PTMs) designed to interact with a target precursor messenger RNA molecule (target pre-mRNA) and mediate a trans­ -splicing reaction resulting in the generation of novel chimeric RNA molecules (chimeric RNA). In particular, the stem cells of the present invention are genetically engineered to express a PTM that will interact with a specific target pre-mRNA expressed within a stem cell as it differentiates so as to result in correction of a genetic defect responsible for a genetic disorder. The methods of the invention encompass transferring a nucleic acid molecule capable of encoding a PTM of interest into a stem cell followed by transplantation of the PTM modified stem cell into a host. As the stem cell differentiates the target pre-mRNA is expressed thereby providing the substrate for a trans -splicing reaction. The present invention is based on the successful transfer and expression of a nucleic acid molecule encoding a PTM capable of interacting with a cystic fibrosis transmembrane conductance regulator (CFTR) pre-mRNA into primary human surface airway progenitor cells. The methods and compositions of the present invention can be used to correct genetic defects associated with a variety of different disorders such as cystic fibrosis, hemophilia, sickle cell anemia, Tay-Sachs disease, thalassemias, polycystic kidney disease and muscular dystrophy, to name a few.
    • 本发明提供通过干细胞中的靶向剪接体介导的转接产生新的核酸分子的方法和组合物。 本发明的组合物包括工程化以表达设计成与靶前体信使RNA分子(靶前体mRNA)相互作用的预转录分子(PTM)的干细胞,并介导导致产生新型嵌合体 RNA分子(嵌合RNA)。 特别地,本发明的干细胞经遗传工程化以表达PTM,其将与干细胞中表达的特异性靶mRNA前体相互作用,因为它会区分,从而导致造成遗传疾病的遗传缺陷的校正 。 本发明的方法包括将能够编码感兴趣的PTM的核酸分子转移到干细胞中,随后将PTM修饰的干细胞移植到宿主中。 当干细胞分化时,靶mRNA前体被表达,从而提供用于转拼反应的底物。 本发明基于能够将能够与囊性纤维化跨膜传导调节子(CFTR)前mRNA相互作用的PTM的核酸分子成功转移和表达进入原代人表面气道祖细胞。 本发明的方法和组合物可用于纠正与各种不同疾病相关的遗传缺陷,例如囊性纤维化,血友病,镰状细胞性贫血,泰萨氏病,地中海贫血,多囊肾病和肌营养不良症,命名为 少数。
    • 62. 发明申请
    • A METHOD OF STRENGTHENING GLASS
    • 加强玻璃的方法
    • WO1992020633A1
    • 1992-11-26
    • PCT/GB1992000913
    • 1992-05-20
    • THE UNIVERSITY OF SHEFFIELDELLIS, BryanCHEN, Xiao, MingSEDDON, Angela, Beth
    • THE UNIVERSITY OF SHEFFIELD
    • C03C17/32
    • C03C17/32C03C17/30C07F7/1804C08F290/144
    • A method of strengthening glass using a mixture of a silane and a resin, the mixture being applied to the glass surface as an aqueous emulsion which is then cured. The silane is a reaction product of a silane coupling agent, which contains an epoxy group, with an unsaturated carboxylic acid, and the resin is a resin having at least two polymerisable unsaturated groups in the molecule. Preferably the silane and the resin have unsaturated reactive groups of matched reactivity. Advantageously the mixture contains about 20 parts of the silane per hundred parts of the resin. In another embodiment a silane coupling agent is dissolved in a polymerisable resin, the mixture of silane and resin is emulsified in water, the emulsion being applied as a coating, which is then cured.
    • 使用硅烷和树脂的混合物强化玻璃的方法,将混合物作为水乳液施加到玻璃表面,然后固化。 硅烷是含有环氧基的硅烷偶联剂与不饱和羧酸的反应产物,树脂是分子中具有至少两个可聚合的不饱和基团的树脂。 优选地,硅烷和树脂具有匹配反应性的不饱和反应性基团。 有利地,该混合物每百份树脂含有约20份硅烷。 在另一个实施方案中,将硅烷偶联剂溶解在可聚合树脂中,硅烷和树脂的混合物在水中乳化,该乳液作为涂层施涂,然后将其固化。