International Journal of Molecular Medical Science, 2024, Vol.14, No.1, 1-7 http://medscipublisher.com/index.php/ijmms 4 Figure 2 Cystic fibrosis In some monogenic genetic diseases, patients may experience a deficiency or insufficient functionality of normal genes. Gene enhancement therapy aims to increase the expression levels of normal genes to achieve therapeutic effects. This method can be implemented by delivering additional normal genes into the patient's body. For instance, through gene enhancement therapy, it is possible to increase the expression of proteins associated with depression-related genes to improve symptoms. 2.2.2 Application in the treatment of polygenic genetic diseases Gene therapy also holds promise in the treatment of polygenic genetic diseases, but compared to single gene inherited diseases, poses more challenges, and research is still in its early stages. Currently, the applications in the treatment of polygenic genetic diseases primarily involve gene silencing therapy, gene regulation therapy, and combination therapy strategies. Gene silencing therapy aims to reduce or eliminate the adverse effects on health by inhibiting the overexpression or production of harmful genes. This can be achieved through RNA interference techniques, such as siRNA or miRNA. By selectively inhibiting the expression of specific genes, it is possible to intervene in the occurrence and progression of polygenic genetic diseases. Polygenic genetic diseases typically involve aberrant regulation of multiple genes. The goal of gene regulation therapy is to restore a normal gene expression pattern by adjusting the balance of the patient's intracellular gene regulatory network. This may involve the use of gene expression regulatory factors (such as transcription factors or nucleases) to modulate the expression levels of relevant genes. The treatment of polygenic genetic diseases often requires the integration of multiple therapeutic strategies. Gene therapy can be combined with other treatment modalities, such as drug therapy, gene editing, or stem cell transplantation, to achieve enhanced therapeutic outcomes. By combining multiple treatment strategies, a more comprehensive intervention in the development and symptoms of polygenic genetic diseases can be achieved. 3 Comparison between Gene Editing and Gene Therapy 3.1 Advantages and limitations in treating genetic diseases The advantages of gene editing include precision; gene editing technologies (such as CRISPR-Cas9) can precisely modify DNA sequences, accurately correcting genetic mutations in patients and restoring normal gene function. Its therapeutic potential is significant; gene editing can potentially cure genetic diseases by repairing or correcting mutations present in the patient's DNA, restoring normal gene expression and function (Li et al., 2017). In terms of persistence, gene editing technology can induce enduring genetic changes in treated cells, which can be passed on to descendant cells, resulting in a long-term therapeutic effect. The limitations of gene editing include safety concerns. Gene editing technology is still in the developmental stage, and unexpected side effects and safety issues, some of which may be unpredictable, could arise. Delivery
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