International Journal of Molecular Medical Science, 2024, Vol.14, No.4, 252-263 http://medscipublisher.com/index.php/ijmms 259 vast, ranging from correcting pathogenic mutations in genes such as APP, PSEN1, and APOE to modulating gene expression to prevent the formation of amyloid-beta plaques and neurofibrillary tangles. Recent studies have demonstrated the ability of CRISPR/Cas9 to reduce amyloid-beta production and improve cognitive function in AD mouse models, paving the way for potential human applications (Rohn et al., 2018). However, the clinical translation of CRISPR/Cas9 remains challenging due to concerns about off-target effects and the need for safe and effective delivery systems to the brain (Bhardwaj et al., 2021). Advances in nanotechnology are contributing to the development of next-generation gene delivery systems that can overcome the limitations of current CRISPR/Cas9 applications in AD. Nanoparticles and other non-viral vectors are being explored as alternatives to traditional viral vectors, offering the potential for safer and more precise delivery of gene-editing tools. These systems can be engineered to cross the blood-brain barrier and deliver CRISPR/Cas9 components directly to the affected regions of the brain, minimizing systemic exposure and reducing the risk of off-target effects (Hanafy et al., 2020). 7.2 Personalized gene therapy Personalized medicine is an emerging trend in gene therapy, particularly in the treatment of complex diseases like AD. By tailoring gene therapy approaches to an individual’s genetic profile, it is possible to achieve more effective and targeted treatments (Zhang, 2024). For instance, individuals with specific mutations in the APP or PSEN1 genes might benefit from therapies designed to correct these mutations using CRISPR/Cas9, while others with different genetic risk factors may require alternative approaches. This level of personalization holds the promise of optimizing therapeutic outcomes and minimizing adverse effects (Paquet et al., 2016; Adji et al., 2022). Another promising direction is the combination of gene therapy with other therapeutic modalities, such as immunotherapy, pharmacotherapy, or lifestyle interventions. Combining CRISPR/Cas9-based gene editing with drugs that target amyloid-beta or tau pathways, for example, could provide a more comprehensive approach to treating AD. Additionally, gene therapy might be used to enhance the efficacy of traditional treatments, such as by increasing the expression of drug targets or by protecting neurons from the toxic effects of amyloid-beta and tau (Barman et al., 2020; Lu et al., 2021). 7.3 Exploration of new therapeutic targets While amyloid-beta and tau have been the primary targets of AD research, emerging evidence suggests that other pathways, such as neuroinflammation and neuroprotection, may also play critical roles in the disease. Gene therapies that target these pathways could provide new avenues for treatment. For example, modulating the expression of genes involved in neuroinflammatory responses or enhancing the production of neuroprotective factors like BDNF could help slow the progression of AD and protect against neuronal loss (Hanafy et al., 2020; Lu et al., 2021). The interplay between genetics, environment, and lifestyle is increasingly recognized as a key factor in the development and progression of AD. Future gene therapy research may focus on understanding how environmental factors such as diet, exercise, and exposure to toxins interact with genetic predispositions to influence disease risk. This knowledge could lead to the development of gene therapies that are not only tailored to an individual’s genetic profile but also take into account their environmental exposures and lifestyle choices, offering a more holistic approach to AD prevention and treatment (Adji et al., 2022). The future of gene therapy for Alzheimer's disease lies in the continued advancement of gene-editing technologies, the development of personalized approaches, and the exploration of new therapeutic targets. These emerging trends hold the promise of transforming the treatment landscape for AD, offering hope for more effective and enduring interventions. 8 Concluding Remarks Gene therapy represents a transformative approach in the treatment of Alzheimer's disease (AD), offering the potential to address the underlying genetic and molecular causes of this complex neurodegenerative disorder. By
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