首页> 外国专利> Haplotype Based Generalizable Allele Specific Silencing for Therapy of Cardiovascular Disease

Haplotype Based Generalizable Allele Specific Silencing for Therapy of Cardiovascular Disease

机译:基于单体型的通用等位基因特异性沉默治疗心血管疾病

摘要

RNAi therapeutic systems in accordance with various embodiments of the invention provide for techniques for silencing expression of deleterious alleles using RNAi therapeutics targeting common variants of alleles. In many embodiments, processes and workflows for identifying common variants of alleles according to repeatedly occurring sets of SNPs are provided. The common variants can be found on genes where deleterious mutations can occur. The common variants can be the basis for targeting with RNAi therapeutics. Thereby, some embodiments of the invention enable efficient and cost saving targeting of common variants using RNAi therapeutics as opposed to individualized deleterious mutation targeting. Several embodiments of the invention further provide for processes for sequencing and phasing subject samples. After sequencing and phasing, some embodiments can apply the common variant targeted RNAi therapeutics to treat deleterious mutations.
机译:根据本发明的各种实施方案的RNAi治疗系统提供了使用靶向等位基因的常见变体的RNAi治疗剂来沉默有害等位基因表达的技术。在许多实施例中,提供了用于根据重复出现的SNP集来识别等位基因的常见变体的过程和工作流。常见变体可以在可能发生有害突变的基因上找到。常见的变体可以作为靶向RNAi治疗剂的基础。因此,与个体化有害突变靶向相反,本发明的一些实施方案能够使用RNAi治疗剂有效且节约成本地靶向常见变体。本发明的几个实施方案还提供了用于对样品进行测序和定相的方法。在测序和定相之后,一些实施方案可以将共同的变体靶向的RNAi治疗剂应用于治疗有害突变。

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