Table 1 Different systems used to modify gene expression and their main advantages and disadvantages. This is the summary of paragraphs that include references from [1] to [145]
From: Current strategies employed in the manipulation of gene expression for clinical purposes
| Â | Options | Key feature | Advantages | Disadvantages |
|---|---|---|---|---|
Genome Editing Nucleases | • MNs • ZFNs • TALENs • CRISPR/Cas | • Locus-specific | • One-time treatment • Versatility and multiplex ability | • Off-target effects leading to mutagenesis and translocations |
Transposons | • SB • PB • TcBuster • Tol2 | • Non locus-specific | • High cloning capacity • Low toxicity • Biosafety | • Transposition efficiency • Insertional mutagenesis |
Episomes | • MAC, HAC • Putative ORI • S/MAR family | • Transient (potentially longer) | • No insertional mutagenesis • Remain extrachromosomal • Low toxicity • High cloning capacity | • Gene silencing due to host gene regulation mechanisms • Transfection efficiency • Mitotic instability |
Small RNA molecules | • siRNA • shRNA • bifunctional shRNA | • Transient | • No insertional mutagenesis • High efficiency • Chemical modifications to reduce off-target effects | • Transient effect |