发明名称 HAPLOTYPE BASED GENERALIZABLE ALLELE SPECIFIC SILENCING FOR THERAPY OF CARDIOVASCULAR DISEASE
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.
申请公布号 WO2016149684(A2) 申请公布日期 2016.09.22
申请号 WO2016US23305 申请日期 2016.03.18
申请人 THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY 发明人 WHEELER, Matthew;ASHLEY, Euan A.;DAINIS, Alexandra
分类号 C12N15/113 主分类号 C12N15/113
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