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Haplotype Based Generalizable Allele Specific Silencing for Therapy of Cardiovascular Disease
Haplotype Based Generalizable Allele Specific Silencing for Therapy of Cardiovascular Disease
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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
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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.
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