Advances in gene editing technology and the carrier delivery system
LIU Su-nan
BAI Wen-jing
LI Pei-shan
LÜ Wan-liang
Abstract：Gene editing refers to a type of biotechnology that can modify,insert or delete specific target genes in the genome of an organism.This article retrospectively describes the basic principles of gene editing,types of gene editing technologies,drug delivery systems for gene editing and their current clinical application status.Main gene editing techniques include zinc-finger nucleases(ZFN),transcription activator-like effector nucleases(TALEN),the CRISPR-Cas nucleases,base editing(BE),and prime editing(PE).Commonly used delivery carriers comprise adenovirus,adeno-associated virus,lentivirus,lipid nanoparticles,polymer nanoparticles,and exosomes,among which recombinant adenovirus,recombinant adeno-associated virus,and lipid nanoparticles are the most widely used carriers.At present,three gene editing therapies have been approved for clinical uses,with a lot of others at the clinical trial phases.These therapies are being applied primarily for the treatment of genetic disorders,cancers,and infectious diseases.As such,gene editing-based drugs are emerging as a transformative therapeutic strategy with the potential to effectively cure a range of intractable diseases.
Keywords：gene editinggene therapyviral vectorsnon-viral vectorsclinical applications
Publication Date：2026-02-15
Online Publishing Date：2026-08-14(First online date of this platform, not the publication date of the document)
Pages：12( 225-236 )