Mar 21, 2020

Allele-specific genome targeting in the development of precision medicine

Theranostics
Junjiao WuYu Tang

Abstract

The CRISPR-based genome editing holds immense potential to fix disease-causing mutations, however, must also handle substantial natural genetic variations between individuals. Previous studies have shown that mismatches between the single guide RNA (sgRNA) and genomic DNA may negatively impact sgRNA efficiencies and lead to imprecise specificity prediction. Hence, the genetic variations bring about a great challenge for designing platinum sgRNAs in large human populations. However, they also provide a promising entry for designing allele-specific sgRNAs for the treatment of each individual. The CRISPR system is rather specific, with the potential ability to discriminate between similar alleles, even based on a single nucleotide difference. Genetic variants contribute to the discrimination capabilities, once they generate a novel protospacer adjacent motif (PAM) site or locate in the seed region near an available PAM. Therefore, it can be leveraged to establish allele-specific targeting in numerous dominant human disorders, by selectively ablating the deleterious alleles. So far, allele-specific CRISPR has been increasingly implemented not only in treating dominantly inherited diseases, but also in research areas such as genome ...Continue Reading

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Mentioned in this Paper

Location
Genomic DNA
Adjacent
Spatio-Temporal Analysis
Pharmaceutical Preparations
Diagnostic Imaging
RNA, Guide
Platinum metallicum, platinum, Homeopathic preparation
Alleles
Area

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