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一种基因载体及其用于治疗雷柏氏先天性黑矇2型疾病的基因治疗药物

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摘要

本发明涉及一种基因治疗载体，该基因治疗载体用于治疗或预防由RPE65基因突变引起的2型Leber先天性黑蒙症(Leber’s congenital amaurosis,LCA2)。本发明的基因治疗载体包含人类视网膜色素上皮细胞主要蛋白65(hRPE65)、启动子及增加基因表达的血管紧张素II型受体内含子1。本发明还涉及包含上述基因治疗载体的药物及组合物。

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公开/公告号CN107287238B

专利类型发明专利
公开/公告日2020-10-16

原文格式PDF
申请/专利权人厦门继景生物技术有限责任公司;
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申请/专利号CN201710230465.X
发明设计人庞继景;
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申请日2017-04-11
分类号C12N15/864(20060101);A61K48/00(20060101);A61K38/17(20060101);A61P27/02(20060101);A61P9/10(20060101);
代理机构31254 上海集信知识产权代理有限公司;
代理人洪玲
地址 361000 中国(福建)自由贸易试验区厦门片区建港路29号海沧国际物流大厦10楼1001单元D0630
入库时间 2022-08-23 11:17:02

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1. 一种基因载体及其用于治疗雷柏氏先天性黑矇2型疾病的基因治疗药物 [P] . 中国专利： CN107287238B . 2020.10.16
2. 一种基因载体及其用于治疗雷柏氏先天性黑矇2型疾病的基因治疗药物 [P] . 中国专利： CN107287238A . 2017-10-24
3. Isolated polynucleotide, methods for identifying or amplifying and genotyping all or part of a polynucleotide, for separating a polypeptide, to obtain an immunospecific antibody, for identifying an agent and a compound between one or more compounds to be tested, for analyzing the biological characteristics of a patient, to prevent or treat in an individual a disease and disorder or disease linked to the presence of polynucleotide in the genome, to increase or decrease activity in a polypeptide patient, to statistically determine relevant associations between at least one snp is a disease or disease resistance and for diagnosing or determining a disease prognosis or disease resistance, recombinant vector, host cell, polypeptide, immunospecific antibody, therapeutic agent, and compound [P] . BR0209097A . 2005-04-19

机译：分离的多核苷酸，鉴定或扩增和多核苷酸的全部或部分基因型，分离多肽，获得免疫特异性抗体，鉴定一种或多种待测化合物之间的试剂和化合物，分析其生物学特性的方法患者，以预防或治疗与基因组中多核苷酸的存在有关的疾病和病症或疾病，以增加或降低多肽患者的活性，以统计学方式确定至少一种snp是疾病或抗病性以及用于诊断或确定疾病预后或抗病性的重组载体，宿主细胞，多肽，免疫特异性抗体，治疗剂和化合物
4. Processes for detecting the presence of alzheimer's disease and mitochondrial origin in an individual and the genetic mutations that cause alzheimer's disease to inhibit the transcription or translation of genes and one or more nucleic acids encoding a mutant cytochrome c oxidase to selectively introduce a conjugated molecule in mitochondria to build a cell line to evaluate a compound for potential utility in the diagnosis of disorders in the treatment of a disorder and in the diagnosis or treatment of diabetes mellitus to prepare a cibrid animal and to determine the presence of a human disease of origin mitochondrial isolated nucleotide sequences probe kit therapeutic composition ribozyme cell line and animal hybrid [P] . BR9507241A . 1997-09-16

机译：用于检测个体中阿尔茨海默氏病和线粒体起源的存在的方法以及导致阿尔茨海默氏病抑制基因和一种或多种编码突变型细胞色素C氧化酶的核酸的转录或翻译的遗传突变，以选择性地将共轭分子引入线粒体建立一种细胞系，以评估该化合物在疾病诊断，疾病治疗以及糖尿病的诊断或治疗中的潜在效用，从而制备出纤巧的动物并确定是否存在人源性线粒体疾病核苷酸序列探针试剂盒治疗组合物核酶细胞系和动物杂种
5. Nucleic acid, nucleic acid sequence, processes using a nucleic acid, using the vector, producing the polipeptu00ecdeo, administering the polipeptu00ecdeo,To treat a disease or disorder in an animal I, to determine if a disease or disorder in an animal I, to produce any polipeptu00ecdeo,Process for identifying, isolating and amplifying a nucleic acid sequence of neublastina,To diagnose or assess for the presence of a disease or disorder or a predisposiu00e7u00e3opara desnvolvu00ea them,To evaluate a polipeptu00ecdeo of neublastina purified or a derivative or fragment of a modulator activity of precedents for activity in treating or preventing a disease.To treat a neurological distu00fabio, using a sequence of one or more nucleic acids, and for identifying a candidate compound that induces a biological effect mediated by neuroblastina, vector,The cell transformed with nucleic acid, polipeptu00ecdeo, cmposiu00e7u00e3o, antibody, synthetic gene, and set. [P] . BR9911908A . 2001-03-27

机译：核酸，核酸序列，使用核酸的过程，使用载体的过程，产生聚肽，施用聚肽以治疗动物I中的疾病或失调，以确定动物中的疾病或失调I，生产任何脂肽，鉴定，分离和扩增神经母细胞核素核酸序列的方法，诊断或评估疾病或病症或它们的易感性，评估纯化的神经母细胞的脂肽或先例的调节剂活性的衍生物或片段，用于治疗或预防疾病。使用一种或多种核酸序列治疗神经系统疾病，并鉴定候选化合物诱导由神经母细胞，载体介导的生物学效应的细胞，该细胞用核酸，lipeptept，cmposi，抗体，合成基因和集合转化。