The Application of CRISPR/Cas9 Technology in Neurological Diseases
DOI:
https://doi.org/10.54097/m3mqzp05Keywords:
Neurological disease; CRISPR/Cas9 system; Huntington's disease; Alzheimer's disease.Abstract
Neurological disease refers to the functional disorders of the central nervous system, peripheral nervous system or plant nervous system caused by a variety of causes such as bacterial infection or genetic. Patients usually show problems such as conscious disorder, sensory disorders, and motor disorder. Common neurological diseases such as Huntington, and Alzheimer's disease, have always been one of the main diseases that endanger human life because of their complex mechanisms and treatment. Gene therapy has always been considered a way to treat diseases. The discovery of Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) has improved gene editing technology and realized the application of various biological fields. CRISPR uses sgRNA to identify target sequences, so that CAS9 protein cuts DNA, and combines the guided RNA with the recognized DNA. Then the nucleic acid-cutting enzyme breaks the DNA dual chain structure, and the host cells were repaired through homologous or non-source mechanisms. CRISPR/Cas9 technology can better perform genetic treatment of neuropathy, making it possible for the development and new treatment methods of human neurodegenerative diseases. This review summarizes the basic principles of CRISPR/Cas9 technology and its application in Huntington's and Alzheimer's disease, as well as discusses the feasibility of CRISPR technology in neurological diseases in the future.
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References
GBD 2015 Neurological Disorders Collaborator Group. Global, regional, and national burden of neurological disorders during 1990-2015: a systematic analysis for the Global Burden of Disease Study 2015. Lancet Neurol. 2017 Nov;16(11):877-897.
2023 Alzheimer's disease facts and figures. Alzheimers Dement. 2023 Apr;19(4):1598-1695.
Mojica FJ, Díez-Villaseñor C, Soria E, Juez G. Biological significance of a family of regularly spaced repeats in the genomes of Archaea, Bacteria and mitochondria. Mol Microbiol. 2000 Apr;36(1):244-6.
Jinek M, Chylinski K, Fonfara I, Hauer M, Doudna JA, Charpentier E. A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science. 2012 Aug 17;337(6096):816-21.
Rohn TT, Kim N, Isho NF, Mack JM. The Potential of CRISPR/Cas9 Gene Editing as a Treatment Strategy for Alzheimer's Disease. J Alzheimers Dis Parkinsonism. 2018;8(3):439.
Duan W, Urani E, Mattson MP. The potential of gene editing for Huntington's disease. Trends Neurosci. 2023 May;46(5):365-376.
Alkanli SS, Alkanli N, Ay A, Albeniz I. CRISPR/Cas9 Mediated Therapeutic Approach in Huntington's Disease. Mol Neurobiol. 2023 Mar;60(3):1486-1498.
Cai L, Fisher AL, Huang H, Xie Z. CRISPR-mediated genome editing and human diseases. Genes Dis. 2016 Aug 30;3(4):244-251.
Kantor A, McClements ME, MacLaren RE. CRISPR-Cas9 DNA Base-Editing and Prime-Editing. Int J Mol Sci. 2020 Aug 28;21(17):6240.
Anzalone AV, Randolph PB, Davis JR, Sousa AA, Koblan LW, Levy JM, Chen PJ, Wilson C, Newby GA, Raguram A, Liu DR. Search-and-replace genome editing without double-strand breaks or donor DNA. Nature. 2019 Dec;576(7785):149-157.
Kumar A, Kumar V, Singh K, Kumar S, Kim YS, Lee YM, Kim JJ. Therapeutic Advances for Huntington's Disease. Brain Sci. 2020 Jan 12;10(1):43.
Tabrizi SJ, Ghosh R, Leavitt BR. Huntingtin Lowering Strategies for Disease Modification in Huntington's Disease. Neuron. 2019 Mar 6;101(5):801-819.
Harper SQ, Staber PD, He X, Eliason SL, Martins IH, Mao Q, Yang L, Kotin RM, Paulson HL, Davidson BL. RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model. Proc Natl Acad Sci U S A. 2005 Apr 19;102(16):5820-5.
Wang G, Liu X, Gaertig MA, Li S, Li XJ. Ablation of huntingtin in adult neurons is nondeleterious but its depletion in young mice causes acute pancreatitis. Proc Natl Acad Sci U S A. 2016 Mar 22;113(12):3359-64.
Shin JW, Kim KH, Chao MJ, Atwal RS, Gillis T, MacDonald ME, Gusella JF, Lee JM. Permanent inactivation of Huntington's disease mutation by personalized allele specific CRISPR/Cas9. Hum Mol Genet. 2016 Oct 15;25(20):4566-4576.
Kolli N, Lu M, Maiti P, Rossignol J, Dunbar GL. CRISPR-Cas9 Mediated Gene-Silencing of the Mutant Huntingtin Gene in an In Vitro Model of Huntington's Disease. Int J Mol Sci. 2017 Apr 2;18(4):754.
Yang S, Chang R, Yang H, Zhao T, Hong Y, Kong HE, Sun X, Qin Z, Jin P, Li S, Li XJ. CRISPR/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of Huntington's disease. J Clin Invest. 2017 Jun 30;127(7):2719-2724.
Hosfield, D.J.; Mol, C.D.; Shen, B.; Tainer, J.A. Structure of the DNA repair and replication endonuclease and exonuclease FEN-1: Coupling DNA and PCNA binding to FEN-1 activity. Cell1998,95, 135-146.
Querfurth HW, LaFerla FM. Alzheimer's disease. N Engl J Med. 2010 Jan 28;362(4):329-44.
Schellenberg GD, Bird TD, Wijsman EM, Orr HT, Anderson L, Nemens E, White JA, Bonnycastle L, Weber JL, Alonso ME, et al. Genetic linkage evidence for a familial Alzheimer's disease locus on chromosome 14. Science. 1992 Oct 23;258(5082):668-71. Lu L, Xi Y, Cai Y, Sun M, Yang H. Application of CRISPR/CAS9 in Alzheimer’s disease. Frontiers in Neuroscience [Internet]. 2021 Dec 21;15.
Ortiz-Virumbrales M, Moreno CL, Kruglikov I, Marazuela P, Sproul A, Jacob S, Zimmer M, Paull D, Zhang B, Schadt EE, Ehrlich ME, Tanzi RE, Arancio O, Noggle S, Gandy S. CRISPR/Cas9-Correctable mutation-related molecular and physiological phenotypes in iPSC-derived Alzheimer's PSEN2 N141I neurons. Acta Neuropathol Commun. 2017 Oct 27;5(1):77.
Rohn TT, Kim N, Isho NF, Mack JM. The Potential of CRISPR/Cas9 Gene Editing as a Treatment Strategy for Alzheimer's Disease. J Alzheimers Dis Parkinsonism. 2018;8(3):439.
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