International Journal of Molecular Medical Science, 2024, Vol.14, No.1, 42-47 http://medscipublisher.com/index.php/ijmms 47 However, despite the enormous potential of gene editing technology in the treatment of neurological diseases, it still faces challenges in delivery efficiency, accuracy, safety, and ethics. It is necessary to address the limitations of gene editing technology in order to achieve its wider application in the treatment of neurological diseases. The key to future development is to improve the efficiency and penetration of delivery systems, enhance the accuracy of editing techniques, and establish strict clinical trial and regulatory frameworks. Through the comprehensive application of various treatment strategies, such as combination therapy, gene editing technology is expected to bring better results to the treatment of neurological diseases. In addition, advances in genomics will provide a better foundation for precise treatment of neurological diseases. In summary, gene editing technology has shown great potential in the treatment of neurological diseases, and future research and development will continue to promote progress in this field. Improving delivery systems, increasing the accuracy of editing techniques, combining treatment strategies, genomics research, and strengthening clinical applications and supervision will promote the further development of gene editing technology in the treatment of neurological diseases, bringing better therapeutic effects to patients. References Amin M., Shahid K., Nasir R., Alam I., and Anwar P., 2018, CRISPR/Cas9: A revolution towards, treatment for neurodegenerative disorders, Open J Biotechnol Bioeng Res. 2(1): 001-008. Bhattacharjee G., Gohil N., Khambhati K., Mani I., Maurya R., Karapurkar J.K., Gohil., Chu D.T., Vu-Thi H., Alzahrani K.J., Show P.L., Rawal R.M., Ramakrishna S., and Singh V., 2022, Current approaches in CRISPR-Cas9 mediated gene editing for biomedical and therapeutic applications, Journal of Controlled Release, 343: 703-723. https://doi.org/10.1016/j.jconrel.2022.02.005 Ekman F.K., Ojala D.S., Adil M.M., Lopez P.A., Schaffer D.V., and Gaj T., 2019, CRISPR-Cas9-mediated genome editing increases lifespan and improves motor deficits in a huntington’s disease mouse model, Molecular Therapy Nucleic Acids, 17: 829-839. https://doi.org/10.1016/j.omtn.2019.07.009 PMid:31465962 PMCid:PMC6717077 Gumusgoz E., Guisso D.R., Kasiri S., Wu J., Dear M., Verhalen B., Nitschke S., Mitra S., Nitschke F., and Minassian B.A., 2021, Targeting Gys1 with AAV‐SaCas9 decreases pathogenic polyglucosan bodies and neuroinflammation in Adult Polyglucosan Body and Lafora disease mouse models, Neurotherapeutics, 18: 1414-1425. https://doi.org/10.1007/s13311-021-01040-7 PMid:33830476 PMCid:PMC8423949 He C.H., Jiang W.Z., Zhang L.W., Ruan M.H., Zhou H.W., and Yu J.R., 2021, Current status and future perspectives of rare disease research, Yichuan (Hereditas(Beijing)), 43(6): 531-544. Hitti F.L., Gonzalez-Alegre P., and Lucas T.H., 2019, Gene therapy for neurologic disease: a neurosurgical review, World Neurosurgery, 121: 261-273. https://doi.org/10.1016/j.wneu.2018.09.097 PMid:30253990 Li X.S., Wang Q.S., and Wang R., 2022, Advantages of CRISPR-Cas9 combined organoid model in the study of congenital nervous system malformations, Front. Bioeng. Biotechnol, 10: 932936. https://doi.org/10.3389/fbioe.2022.932936 PMid:36118578 PMCid:PMC9478582 Ma B.X., Shen W.L., Wang X., Li Z., and Xu K., 2020, Gene edited animal models applied in human disease research, Shengwu Gongcheng Xuebao (Chinese Journal of Biotechnology), 36(5): 849-860. Pazzaglia S., and Pioli C., 2019, Multifaceted role of PARP-1 in DNA repair and inflammation: pathological and therapeutic implications in cancer and non-cancer diseases, Cells, 9(1): 41. https://doi.org/10.3390/cells9010041 Shi M.G., Shen Z.Y., Zhang N., Wang L.Y., Yu C.Y., and Yang Z., 2021, CRISPR/Cas9 technology in disease research and therapy: a review. Chinese Journal of Biotechnology, 37(4): 1205-1228. Song S.Z., Lu R., Zhang T., He Z.Y., Wu Z.M.Q., Cheng Y., and Zhou M.M., 2020, Research progress of CRISPR /Cas9 gene editing technology in goat and sheep, Biotechnology Bulletin, 36(3): 62-68. Zhu X.C, Xie X.Y., Zhao X.R., Xu L.N., He Z.Y., and Zhou W., 2023, Construction and characterization of mice with conditional knockout of Stat3 gene in microglia, Shanghai Jiaotong Daxue Xuebao (Journal of Shanghai Jiao Tong University (Medical Science)), 43(6): 689-698.
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