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Abstract
Gene-based therapy is a crucial approach in precision medicine that targets specific genes in specific cells.
- Key challenges for clinical applications of gene-based therapy include safety, stability, selectivity, delivery, and immune responses.
- Current nucleic acid drugs in clinical trials and on the market include antisense oligonucleotides, siRNA, aptamers, and plasmid nucleic acids.
- Chemical modifications of nucleic acid drugs may enhance their effectiveness and delivery systems for therapeutic use.
- Nanotechnology methods are being explored to improve the delivery of nucleic acid drugs in vivo.
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