CRISPR/Cas9技术在更好地理解和治疗神经系统疾病方面的进展。
Advancement in CRISPR/Cas9 Technology to Better Understand and Treat Neurological Disorders.
作者信息
Datta Aishika, Sarmah Deepaneeta, Kaur Harpreet, Chaudhary Antra, Vadak Namrata, Borah Anupom, Shah Sudhir, Wang Xin, Bhattacharya Pallab
机构信息
Department of Pharmacology and Toxicology, National Institute of Pharmaceutical Education and Research (NIPER), Ahmedabad, Gandhinagar, Gujarat, 382355, India.
Cellular and Molecular Neurobiology Laboratory, Department of Life Science and Bioinformatics, Assam University, Silchar, Assam, 788011, India.
出版信息
Cell Mol Neurobiol. 2023 Apr;43(3):1019-1035. doi: 10.1007/s10571-022-01242-3. Epub 2022 Jun 25.
Neurological disorders have complicated pathophysiology that may involve several genetic mutations. Conventional treatment has limitations as they only treat apparent symptoms. Although, personalized medicine is emerging as a promising neuro-intervention, lack of precision is the major pitfall. Clustered regularly interspaced short palindromic repeat (CRISPR)/Cas9 system is evolving as a technological platform that may overcome the therapeutic limitations towards precision medicine. In the future, targeting genes in neurological disorders may be the mainstay of modern therapy. The present review on CRISPR/Cas9 and its application in various neurological disorders may provide a platform for its future clinical relevance towards developing precise and personalized medicine.
神经疾病具有复杂的病理生理学，可能涉及多种基因突变。传统治疗存在局限性，因为它们仅治疗明显症状。尽管个性化医疗正成为一种有前景的神经干预手段，但缺乏精准性是其主要缺陷。成簇规律间隔短回文重复序列（CRISPR）/Cas9系统正在发展成为一个技术平台，有望克服精准医疗的治疗局限性。未来，针对神经疾病中的基因进行靶向治疗可能成为现代治疗的主流。本综述关于CRISPR/Cas9及其在各种神经疾病中的应用，可能为其未来在开发精准和个性化药物方面的临床相关性提供一个平台。
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