CRISPR-Cas9: A Promising Genome Editing Therapeutic Tool for Alzheimer's Disease-A Narrative Review

被引:37
作者
Barman, Nirmal Chandra [1 ]
Khan, Md. Niuz Morshed [2 ]
Islam, Maidul [3 ]
Nain, Zulkar [1 ]
Roy, Rajib Kanti [4 ]
Haque, Md. Anwarul [1 ]
Barman, Shital Kumar [5 ]
机构
[1] Islamic Univ, Fac Biol Sci, Dept Biotechnol & Genet Engn, Kushtia 7003, Bangladesh
[2] Khulna Univ, Biotechnol & Genet Engn Discipline, Khulna 9208, Bangladesh
[3] Mawlana Bhashani Sci & Technol Univ, Dept Biotechnol & Genet Engn, Tangail 1902, Bangladesh
[4] Jashore Univ Sci & Technol, Dept Nutr & Food Technol, Jashore 7408, Bangladesh
[5] Western Sydney Univ, Sch Sci, Locked Bag 1797, Penrith, NSW 2751, Australia
关键词
Alzheimer's disease (AD); Clinical trial; CRISPR-Cas9; Genome editing; Therapeutic tool; NEUROPATHOLOGIC ASSESSMENT; NATIONAL INSTITUTE; MOUSE MODELS; GENE; CAS9; DELIVERY; MUTATION; RNA; DNA; ASSOCIATION;
D O I
10.1007/s40120-020-00218-z
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
Alzheimer's disease (AD) is a chronic and irreversible neurodegenerative disorder characterized by cognitive deficiency and development of amyloid-beta (A beta) plaques and neurofibrillary tangles, comprising hyperphosphorylated tau. The number of patients with AD is alarmingly increasing worldwide; currently, at least 50 million people are thought to be living with AD. The mutations or alterations in amyloid-beta precursor protein (APP), presenilin-1 (PSEN1), or presenilin-2 (PSEN2) genes are known to be associated with the pathophysiology of AD. Effective medication for AD is still elusive and many gene-targeted clinical trials have failed to meet the expected efficiency standards. The genome editing tool clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 has been emerging as a powerful technology to correct anomalous genetic functions and is now widely applied to the study of AD. This simple yet powerful tool for editing genes showed the huge potential to correct the unwanted mutations in AD-associated genes such asAPP,PSEN1, andPSEN2. So, it has opened a new door for the development of empirical AD models, diagnostic approaches, and therapeutic lines in studying the complexity of the nervous system ranging from different cell types (in vitro) to animals (in vivo). This review was undertaken to study the related mechanisms and likely applications of CRISPR-Cas9 as an effective therapeutic tool in treating AD.
引用
收藏
页码:419 / 434
页数:16
相关论文
共 98 条
[1]   Nanoparticles-mediated CRISPR/Cas9 delivery: Recent advances in cancer treatment [J].
Aghamiri, Shahin ;
Talaei, Sam ;
Ghavidel, Afshin Abdi ;
Zandsalimi, Farshid ;
Masoumi, Saeid ;
Hafshejani, Nahid Heidari ;
Jajarmi, Vahid .
JOURNAL OF DRUG DELIVERY SCIENCE AND TECHNOLOGY, 2020, 56
[2]   Genetics of Alzheimer's Disease [J].
Alonso Vilatela, Maria Elisa ;
Lopez-Lopez, Marisol ;
Yeseas-Gomez, Petra .
ARCHIVES OF MEDICAL RESEARCH, 2012, 43 (08) :622-631
[3]   Search-and-replace genome editing without double-strand breaks or donor DNA [J].
Anzalone, Andrew V. ;
Randolph, Peyton B. ;
Davis, Jessie R. ;
Sousa, Alexander A. ;
Koblan, Luke W. ;
Levy, Jonathan M. ;
Chen, Peter J. ;
Wilson, Christopher ;
Newby, Gregory A. ;
Raguram, Aditya ;
Liu, David R. .
NATURE, 2019, 576 (7785) :149-+
[4]   CRISPR provides acquired resistance against viruses in prokaryotes [J].
Barrangou, Rodolphe ;
Fremaux, Christophe ;
Deveau, Helene ;
Richards, Melissa ;
Boyaval, Patrick ;
Moineau, Sylvain ;
Romero, Dennis A. ;
Horvath, Philippe .
SCIENCE, 2007, 315 (5819) :1709-1712
[5]   Genetic insights in Alzheimer's disease [J].
Bettens, Karolien ;
Sleegers, Kristel ;
Van Broeckhoven, Christine .
LANCET NEUROLOGY, 2013, 12 (01) :92-104
[6]   Current concepts - Memory dysfunction [J].
Budson, AE ;
Price, BH .
NEW ENGLAND JOURNAL OF MEDICINE, 2005, 352 (07) :692-699
[7]  
CARR DB, 1997, AM J MED, V103, P3
[8]   Genetic variation in PCDH11X is associated with susceptibility to late-onset Alzheimer's disease [J].
Carrasquillo, Minerva M. ;
Zou, Fanggeng ;
Pankratz, V. Shane ;
Wilcox, Samantha L. ;
Ma, Li ;
Walker, Louise P. ;
Younkin, Samuel G. ;
Younkin, Curtis S. ;
Younkin, Linda H. ;
Bisceglio, Gina D. ;
Ertekin-Taner, Nilufer ;
Crook, Julia E. ;
Dickson, Dennis W. ;
Petersen, Ronald C. ;
Graff-Radford, Neill R. ;
Younkin, Steven G. .
NATURE GENETICS, 2009, 41 (02) :192-198
[9]   Identification of preexisting adaptive immunity to Cas9 proteins in humans [J].
Charlesworth, Carsten T. ;
Deshpande, Priyanka S. ;
Dever, Daniel P. ;
Camarena, Joab ;
Lemgart, Viktor T. ;
Cromer, M. Kyle ;
Vakulskas, Christopher A. ;
Collingwood, Michael A. ;
Zhang, Liyang ;
Bode, Nicole M. ;
Behlke, Mark A. ;
Dejene, Beruh ;
Cieniewicz, Brandon ;
Romano, Rosa ;
Lesch, Benjamin J. ;
Gomez-Ospina, Natalia ;
Mantri, Sruthi ;
Pavel-Dinu, Mara ;
Weinberg, Kenneth I. ;
Porteus, Matthew H. .
NATURE MEDICINE, 2019, 25 (02) :249-+
[10]   CRISPR-Cas9 for cancer therapy: Opportunities and challenges [J].
Chen, Minjiang ;
Mao, Aiwu ;
Xu, Min ;
Weng, Qiaoyou ;
Mao, Jianting ;
Ji, Jiansong .
CANCER LETTERS, 2019, 447 :48-55