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SYSTEM DELIVERY AND REGULATED EXPRESSION OF PARACRIN GENES FOR TREATMENT OF CARDIOVASCULAR AND OTHER DISEASES

机译:派拉克林基因的系统传递和调控表达,可治疗心血管疾病和其他疾病

摘要

1. A method of treating, improving, or protecting (protecting) an individual or patient from a disease, infection, or condition sensitive to an elevated or stable level of a paracrine peptide or polypeptide in vivo, comprising: (a) (i) (i) providing a nucleic acid or a gene encoding a paracrine polypeptide operably linked to a transcriptional regulatory sequence; or an expression agent, vector, recombinant virus or equivalent, comprising a nucleic acid encoding a paracrine factor or gene, or a nucleic acid expressing a paracrine polypeptide, transcript or mRNA, the expression agent, vector, recombinant virus, or equivalent, may express an encoding paracrine factor nucleic acid, gene, transcript or mRNA in a cell or in vivo; and (ii) introducing or delivering a paracrine polypeptide of a nucleic acid, gene, transcript or mRNA operably linked to a transcription regulatory sequence, or an expression agent, vector, recombinant virus or equivalent, in a cell or individual or patient in need thereof, and by treatment, improvement or protection (protection) of an individual or patient from a disease, infection or condition sensitive to an elevated or stable level of a paracrine polypeptide; (b) cn person (a) according to which the expression agent, vector, recombinant virus or equivalent is or contains: adeno-associated virus (AAV), lentiviral vector or adenovirus vector, AAV serotype AAV5, AAV6, AAV8 or AAV9, originating from rhesus monkey AAV or poppy-derived
机译:1.一种治疗,改善或保护(保护)个体或患者免受体内对旁分泌肽或多肽水平升高或稳定的疾病,感染或病状的方法,该方法包括:(a)(i) (i)提供可操作地连接至转录调控序列的编码旁分泌多肽的核酸或基因;包含表达旁分泌因子或基因的核酸或表达旁分泌多肽,转录物或mRNA的核酸的表达剂,载体,重组病毒或等同物,表达剂,载体,重组病毒或等同物可以表达细胞或体内的编码旁分泌因子的核酸,基因,转录本或mRNA; (ii)在需要其的细胞或个体或患者中引入或递送与转录调控序列可操作连接的核酸,基因,转录物或mRNA的旁分泌多肽,或表达剂,载体,重组病毒或等同物以及通过治疗,改善或保护(保护)个人或患者免受对旁分泌多肽升高或稳定水平敏感的疾病,感染或状况; (b)人(a)所述表达剂,载体,重组病毒或等同物所依据的或含有的:腺相关病毒(AAV),慢病毒载体或腺病毒载体,AAV血清型AAV5,AAV6,AAV8或AAV9,起源于来自恒河猴AAV或罂粟来源

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