AI Article Synopsis
- Gene-based therapy involves modifying gene expression in specific cells to treat diseases using various types of nucleic acids like DNA and RNA.
- Due to their large size and negative charge, effective delivery of these nucleic acids often requires carriers or vectors.
- Recent developments in material sciences and nanotechnology have led to innovative non-viral delivery systems that are currently being tested in clinical trials, showcasing advancements in gene-based therapy.
Article Abstract
Gene-based therapy is the intentional modulation of gene expression in specific cells to treat pathological conditions. This modulation is accomplished by introducing exogenous nucleic acids such as DNA, mRNA, small interfering RNA (siRNA), microRNA (miRNA) or antisense oligonucleotides. Given the large size and the negative charge of these macromolecules, their delivery is typically mediated by carriers or vectors. In this Review, we introduce the biological barriers to gene delivery in vivo and discuss recent advances in material sciences, nanotechnology and nucleic acid chemistry that have yielded promising non-viral delivery systems, some of which are currently undergoing testing in clinical trials. The diversity of these systems highlights the recent progress of gene-based therapy using non-viral approaches.
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| http://dx.doi.org/10.1038/nrg3763 | DOI Listing |
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