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Translational potential of base-editing tools for gene therapy of monogenic diseases

Front Bioeng Biotechnol. 2022-08; 
Vasiliy V Reshetnikov, Angelina V Chirinskaite, Julia V Sopova, Roman A Ivanov, Elena I Leonova
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Codon Optimization GenScript codon usage was named BE4max and had 1.8-fold higher editing efficiency as compared to bisbpNLS BE4 with IDT codons… fold, and GenScript codon optimization yielded 1.3… Get A Quote

摘要

Millions of people worldwide have rare genetic diseases that are caused by various mutations in DNA sequence. Classic treatments of rare genetic diseases are often ineffective, and therefore great hopes are placed on gene-editing methods. A DNA base-editing system based on nCas9 (Cas9 with a nickase activity) or dCas9 (a catalytically inactive DNA-targeting Cas9 enzyme) enables editing without double-strand breaks. These tools are constantly being improved, which increases their potential usefulness for therapies. In this review, we describe the main types of base-editing systems and their application to the treatment of monogenic diseases in experiments and . Additionally, to understand the therapeutic potent... More

关键词

base editing, dCas9, gene therapy, monogenic disease, nCas9, prime editor
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