Cancer as a genetic disorder is one of the leading causes of death worldwide. Conventional anticancer options
such as chemo- and/or radio-therapy have their own drawbacks and could not provide a cure in most cases at present.
More effective therapeutic strategies with less side effects are urgently needed. Aptamers, also known as chemical antibodies,
are single strand DNA or RNA molecules that can bind to their target molecules with high affinity and specificity.
Such site-specific binding ability of aptamers facilitates the delivery and interaction of exogenous nucleic acids with diseased
genes. Thus, aptamer-guided gene therapy has emerged as a promising anticancer strategy in addition to the classic
treatment regimen. Aptamers can directly deliver anti-cancer nucleic acids, e.g. small interfering RNA, micro RNA, antimicroRNA
and small hairpin RNA, to cancer cells or function as a targeting ligand to guide nanoparticles containing
therapeutic nucleic acids. This review focuses on recent progress in aptamer-mediated gene therapy for the treatment of
hepatocellular carcinoma and other types of cancers, shedding light on the potential of this novel approach of targeted
cancer gene therapy.
Keywords: Aptamer, gene therapy, miRNA, RNAi, cancer, hepatocellular carcinoma.
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