Translational potential of base-editing tools for gene therapy of monogenic diseases.
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ABSTRACT:
SUBMITTER: Reshetnikov VV
PROVIDER: S-EPMC9399415 | biostudies-literature | 2022
REPOSITORIES: biostudies-literature
Publications
Translational potential of base-editing tools for gene therapy of monogenic diseases.
Reshetnikov Vasiliy V VV Chirinskaite Angelina V AV Sopova Julia V JV Ivanov Roman A RA Leonova Elena I EI
Frontiers in bioengineering and biotechnology 20220810
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 useful ...[more]
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