ScholarWorks@중앙대학교

초록

CRISPR-Cas technologies have emerged as powerful and versatile tools in gene therapy. Inaddition to the widely used SpCas9 system, alternative platforms including modified aminoacid sequences, size-optimized variants, and other Cas enzymes from diverse bacterial specieshave been developed to apply this technology in various genetic contexts. In addition,base editors and prime editors for precise gene editing, the Cas13 system targeting RNA,and CRISPRa/i systems have enabled diverse and adaptable approaches for genome andRNA editing, as well as for regulating gene expression. Typically, CRISPR-Cas components aretransported to the target in the form of DNA, RNA, or ribonucleoprotein complexes usingvarious delivery methods, such as electroporation, adeno-associated viruses, and lipidnanoparticles. To amplify therapeutic efficiency, continued developments in targeted deliverytechnologies are required, with increased safety and stability of therapeutic biomolecules. CRISPR-based therapeutics hold an inexhaustible potential for the treatment of manydiseases, including rare congenital diseases, by making permanent corrections at the genomicDNA level. In this review, we present various CRISPR-based tools, their delivery systems,and clinical progress in the CRISPR-Cas technology, highlighting its innovative prospectsfor gene therapy.

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