AI Article Synopsis
- Allele-specific silencing by RNA interference (ASP-RNAi) selectively targets disease-causing alleles without impacting normal alleles, making it a promising therapeutic approach.
- Designing effective small interfering RNAs (siRNAs) for ASP-RNAi is challenging due to the lack of reliable measures for determining their effectiveness.
- The authors introduced two new formulas for calculating an "ASP-score," which helps in unbiasedly identifying optimal siRNAs for allele discrimination, enhancing the development of allele-specific therapies.
Article Abstract
Allele-specific silencing by RNA interference (ASP-RNAi) is an atypical RNAi that is capable of discriminating target alleles from non-target alleles, and may be therapeutically useful for specific inhibition of disease-causing alleles without affecting their corresponding normal alleles. However, it is difficult to design and select small interfering RNA (siRNAs) that confer ASP-RNAi. A major problem is that there are few appropriate measures in determining optimal allele-specific siRNAs. Here we show two novel formulas for calculating a new measure of allele-discrimination, named "ASP-score". The formulas and ASP-score allow for an unbiased determination of optimal siRNAs, and may contribute to characterizing such allele-specific siRNAs.
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| http://dx.doi.org/10.1007/s11033-014-3586-7 | DOI Listing |
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