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CRISPR-Cas9-Mediated Gene Therapy in Neurological Disorders

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Abstract

Neurological disorders are primarily diseases with sophisticated etiology that are always refractory and recrudescent. The major obstruction to effective therapies for neurological disorders is the poor understanding of their pathogenic mechanisms. CRISPR-Cas9 technology, which allows precise and effective gene editing in almost any cell type and organism, is accelerating the pace of basic biological research. An increasing number of groups are focusing on uncovering the molecular mechanisms of neurological disorders and developing novel therapies using the CRISPR-Cas9 system. This review highlights the application of CRISPR-Cas9 technology in the treatment of neurological disorders, including Alzheimer’s disease, amyotrophic lateral sclerosis and/or frontotemporal dementia, Duchenne muscular dystrophy, Dravet syndrome, epilepsy, Huntington’s disease, and Parkinson’s disease. Hopefully, it will improve our understanding of neurological disorders and give insights into future treatments for neurological disorders.

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Acknowledgements

We would like to thank Dr. Zhenyu Hao (Zhengzhou University of Light Industry) for his comments and suggestions.

Funding

This work was supported by grants from the National Natural Science Foundation of China (81801127, 81771226, 81800792, U1804186), Natural Science Foundation of Henan Province for Distinguished Young Scholars (202300410307), Science and Technology Innovative Research Team in Higher Educational Institutions of Henan Province (19IRTSTHN003), Henan Province Science and Technology Project (212102310215), Major Cultivation Plan of Scientific and Technological Achievements from Natural Science Class of Xinxiang Medical University (20172DCG-03), Key Scientific Research Program of Higher Education in Henan Province (21A180023), Xinxiang Scientific and Technological Project (GG2019009), Doctoral Scientific Research Program Foundation of Xinxiang Medical University (XYBSKYZZ201523), and Open Program of Henan Key Lab of Biological Psychiatry (ZDSYS2015004).

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Authors and Affiliations

  1. School of Life Science and Technology, Henan Mental Hospital, Xinxiang Medical University, Number 601, Jinsui Road, Xinxiang, 453003, Henan, China

    Lihong Guan, Ciqing Yang, Han Li & Juntang Lin

  2. Henan Key Laboratory of Medical Tissue Regeneration, Xinxiang, 453003, Henan, China

    Lihong Guan, Ciqing Yang, Jiang Du, Han Li & Juntang Lin

  3. Henan Joint International Research Laboratory of Stem Cell Medicine, Stem Cell and Biotherapy Engineering Research Center of Henan, Xinxiang, 453003, Henan, China

    Lihong Guan, Ciqing Yang, Jiang Du, Han Li & Juntang Lin

  4. Henan Key Lab of Biological Psychiatry, Henan International Joint Laboratory of Psychiatry and Neuroscience, Xinxiang, 453003, Henan, China

    Lihong Guan

  5. College of Food and Bioengineering, Zhengzhou University of Light Industry, Zhengzhou, 450002, Henan, China

    Yawei Han

  6. Medical College of Pingdingshan University, Pingdingshan, 467000, Henan, China

    Suxiang Lu

  7. School of Medical Engineering, Xinxiang Medical University, Xinxiang, 453003, Henan, China

    Jiang Du & Juntang Lin

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  1. Lihong Guan
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Contributions

Both GLH and LJT conceived the idea for the article. Both GLH and HYW searched the literature. GLH wrote the manuscript. LSX created the figures with Adobe Illustrator. YCQ, DJ, and LH revised the manuscript. LJT supervised the manuscript.

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Correspondence to Juntang Lin.

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Guan, L., Han, Y., Yang, C. et al. CRISPR-Cas9-Mediated Gene Therapy in Neurological Disorders. Mol Neurobiol 59, 968–982 (2022). https://doi.org/10.1007/s12035-021-02638-w

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