International Journal of Molecular Medical Science, 2024, Vol.14, No.1, 8-15 http://medscipublisher.com/index.php/ijmms 11 in the treatment of leukemia. Gene therapy has also shown improvement in ocular diseases like retinal pigmentosa and hereditary optic neuropathy. However, gene therapy also faces limitations and challenges. Delivery efficiency remains a challenge, and improvements are needed to effectively deliver therapeutic genes to target cells and tissues. Safety is another crucial consideration, with attention required for immune reactions, inflammatory responses, or potential adverse events. Ensuring stable expression and persistence of the therapeutic gene, as well as addressing individual variability, are also issues that need resolution. Additionally, the high cost of gene therapy restricts its widespread application, and accessibility is further limited by technical requirements and healthcare resource constraints. Moreover, gene therapy involves ethical and moral considerations, such as privacy protection and fair distribution. Despite the challenges and limitations, gene therapy is a promising field that provides new options for the treatment of many diseases. With advances in scientific technology and accumulated experience, it is believed that gene therapy will continue to evolve in the future, bringing benefits to more patients. 2.3 Breakthrough of CRISPR-Cas9 technology and comparison with traditional gene therapy approaches CRISPR-Cas9 technology is a revolutionary gene editing tool that offers numerous breakthrough advantages compared to traditional gene therapy methods. CRISPR-Cas9 enables precise gene editing in the genome, allowing for gene knockout, repair, and insertion operations. In contrast, traditional gene therapy typically involves gene addition or replacement. CRISPR-Cas9 is characterized by its flexibility and versatility, making it widely applicable in both research and treatment. This technology achieves targeted editing of specific DNA sequences by designing guide RNA, enhancing treatment specificity-a feature often lacking in traditional gene therapy methods. CRISPR-Cas9 technology can directly perform gene editing in vivo, enabling patients to receive treatment without the need for ex vivo cells or gene delivery vectors, thereby enhancing treatment efficiency and convenience (Chen et al., 2019). While CRISPR-Cas9 technology faces some challenges and safety risks, it is considered to have groundbreaking potential, opening up new possibilities in the field of gene therapy. It provides a more precise, flexible, and efficient approach to gene therapy, offering promising prospects for the treatment of genetic diseases and advancements in biological research. As this technology continues to develop and improve, it is believed to bring forth further innovations and progress in the field of gene therapy. 3 CRISPR-Cas9 Technology in the Prospects of Gene Therapy 3.1 Application in the treatment of monogenic genetic diseases CRISPR-Cas9 technology has shown promising progress in the treatment of monogenic genetic diseases (Xu, 2020). Cystic fibrosis, a common genetic disease, can be corrected using CRISPR-Cas9 technology to rectify mutations in the human CFTR gene, restoring its function, which has been successful in both in vitro and in vivo studies. Similarly, for certain genetic blood disorders such as sickle cell anemia and β-thalassemia, CRISPR-Cas9 technology can be applied to correct patients' hematopoietic stem cells, preventing or alleviating disease symptoms. Additionally, immune deficiency disorders like severe combined immunodeficiency can be addressed by using CRISPR-Cas9 technology for gene editing to restore patients' immune function. For neurological disorders such as Huntington's disease, amyotrophic lateral sclerosis, and hereditary retinal dystrophy (Figure 2), CRISPR-Cas9 is also being researched for potential therapeutic applications. While the application in the treatment of monogenic genetic diseases faces some challenges, CRISPR-Cas9-based therapies are still regarded as a highly promising field, offering the potential for more effective and personalized treatment options for patients
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