{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,19]],"date-time":"2026-10-19T09:42:13Z","timestamp":1792402933746,"version":"4.4.0"},"reference-count":98,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2022,9,1]],"date-time":"2022-09-01T00:00:00Z","timestamp":1661990400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2022,9,1]],"date-time":"2022-09-01T00:00:00Z","timestamp":1661990400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2021,7,3]],"date-time":"2021-07-03T00:00:00Z","timestamp":1625270400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100014555","name":"Murdoch Children's Research Institute","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100014555","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001407","name":"Department of Biotechnology, Ministry of Science and Technology, India","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100001407","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001409","name":"Department of Science and Technology, Ministry of Science and Technology, India","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100001409","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100006013","name":"United Arab Emirates University","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100006013","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100007449","name":"Takeda Science Foundation","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100007449","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Journal of Advanced Research"],"published-print":{"date-parts":[[2022,9]]},"DOI":"10.1016\/j.jare.2021.07.001","type":"journal-article","created":{"date-parts":[[2021,7,6]],"date-time":"2021-07-06T00:47:04Z","timestamp":1625532424000},"page":"207-221","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":131,"special_numbering":"C","title":["CRISPR\/Cas9 gene editing: New hope for Alzheimer's disease therapeutics"],"prefix":"10.1016","volume":"40","author":[{"given":"Shanu","family":"Bhardwaj","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kavindra Kumar","family":"Kesari","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mahesh","family":"Rachamalla","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shalini","family":"Mani","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ghulam Md.","family":"Ashraf","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Saurabh Kumar","family":"Jha","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pravir","family":"Kumar","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rashmi K.","family":"Ambasta","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Harish","family":"Dureja","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hari Prasad","family":"Devkota","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gaurav","family":"Gupta","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dinesh Kumar","family":"Chellappan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sachin Kumar","family":"Singh","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kamal","family":"Dua","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Janne","family":"Ruokolainen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mohammad Amjad","family":"Kamal","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shreesh","family":"Ojha","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Niraj Kumar","family":"Jha","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"issue":"2","key":"10.1016\/j.jare.2021.07.001_b0005","doi-asserted-by":"crossref","first-page":"341","DOI":"10.3233\/JAD-140933","article-title":"Tau phosphorylation, molecular chaperones, and ubiquitin E3 ligase: clinical relevance in Alzheimer's disease","volume":"43","author":"Kumar","year":"2015","journal-title":"J Alzheimers Dis"},{"issue":"3","key":"10.1016\/j.jare.2021.07.001_b0010","doi-asserted-by":"crossref","first-page":"440","DOI":"10.1016\/j.trci.2017.07.001","article-title":"Neuropsychiatric signs and symptoms of Alzheimer's disease: New treatment paradigms","volume":"3","author":"Lanct\u00f4t","year":"2017","journal-title":"Alzheimers Dement (N Y)"},{"key":"10.1016\/j.jare.2021.07.001_b0015","doi-asserted-by":"crossref","unstructured":"Pandey G, Ramakrishnan V. Invasive and non-invasive therapies for Alzheimer's disease and other amyloidosis. Biophys Rev. 2020 Oct;12(5):1175-1186. doi: 10.1007\/s12551-020-00752-y. Epub 2020 Sep 15. PMID: 32930962; PMCID: PMC7575678.","DOI":"10.1007\/s12551-020-00752-y"},{"issue":"12","key":"10.1016\/j.jare.2021.07.001_b0020","doi-asserted-by":"crossref","DOI":"10.1098\/rsob.200286","article-title":"Alzheimer's disease-like perturbations in HIV-mediated neuronal dysfunctions: understanding mechanisms and developing therapeutic strategies","volume":"10","author":"Jha","year":"2020","journal-title":"Open Biol"},{"issue":"2","key":"10.1016\/j.jare.2021.07.001_b0025","doi-asserted-by":"crossref","first-page":"113","DOI":"10.1111\/jnc.14687","article-title":"Nuclear factor-kappa \u03b2 as a therapeutic target for Alzheimer's disease","volume":"150","author":"Jha","year":"2019","journal-title":"J Neurochem"},{"issue":"4","key":"10.1016\/j.jare.2021.07.001_b0030","doi-asserted-by":"crossref","first-page":"891","DOI":"10.3233\/JAD-150379","article-title":"Impact of Insulin Degrading Enzyme and Neprilysin in Alzheimer's Disease Biology: Characterization of Putative Cognates for Therapeutic Applications","volume":"48","author":"Jha","year":"2015","journal-title":"J Alzheimers Dis"},{"issue":"10","key":"10.1016\/j.jare.2021.07.001_b0035","doi-asserted-by":"crossref","first-page":"795","DOI":"10.2174\/1568026619666190412121811","article-title":"ABC Transporters in Neurological Disorders: An Important Gateway for Botanical Compounds Mediated Neuro-Therapeutics","volume":"19","author":"Jha","year":"2019","journal-title":"Curr Top Med Chem"},{"issue":"1","key":"10.1016\/j.jare.2021.07.001_b0040","doi-asserted-by":"crossref","first-page":"15","DOI":"10.3233\/JAD-170589","article-title":"Hypoxia-Induced Signaling Activation in Neurodegenerative Diseases: Targets for New Therapeutic Strategies","volume":"62","author":"Jha","year":"2018","journal-title":"J Alzheimers Dis"},{"issue":"4","key":"10.1016\/j.jare.2021.07.001_b0045","doi-asserted-by":"crossref","first-page":"1017","DOI":"10.3233\/JAD-160623","article-title":"Stress-Induced Synaptic Dysfunction and Neurotransmitter Release in Alzheimer's Disease: Can Neurotransmitters and Neuromodulators be Potential Therapeutic Targets?","volume":"57","author":"Jha","year":"2017","journal-title":"J Alzheimers Dis"},{"key":"10.1016\/j.jare.2021.07.001_b0050","first-page":"321","article-title":"Alzheimer\u2019s disease facts and figures","volume":"15","author":"Gaugler","year":"2019","journal-title":"Alzheimers Dement"},{"key":"10.1016\/j.jare.2021.07.001_b0055","doi-asserted-by":"crossref","first-page":"37","DOI":"10.1186\/alzrt269","article-title":"Alzheimer\u2019s disease drug-development pipeline: Few candidates, frequent failures","volume":"6","author":"Cummings","year":"2014","journal-title":"Alzheimers Res Ther"},{"key":"10.1016\/j.jare.2021.07.001_b0060","doi-asserted-by":"crossref","first-page":"319","DOI":"10.1016\/S1474-4422(13)70276-X","article-title":"Targeting the secretase BACE1 for Alzheimer\u2019s disease therapy","volume":"13","author":"Yan","year":"2014","journal-title":"Lancet Neurol"},{"key":"10.1016\/j.jare.2021.07.001_b0065","doi-asserted-by":"crossref","unstructured":"\u0160im\u00edcG,Bab\u00edcLeko M, Wray S, Harrington C,DelalleI,Jovanov-Milo\u0161ev\u00edcN,Ba\u017eadona, D,Bu\u00e9e L, De Silva R, Di Giovanni G, et al. Tau protein hyperphosphorylation and aggregation in Alzheimer\u2019s disease and other tauopathies, and possible neuroprotective strategies. Biomolecules 2016;6:6.","DOI":"10.3390\/biom6010006"},{"key":"10.1016\/j.jare.2021.07.001_b0070","article-title":"Cerebral microvascular accumulation of tau oligomers in Alzheimer\u2019s disease and related tauopathies","author":"Castillo-Carranza","year":"20178257","journal-title":"Aging Dis"},{"key":"10.1016\/j.jare.2021.07.001_b0075","doi-asserted-by":"crossref","first-page":"409","DOI":"10.1111\/jnc.14011","article-title":"Microtubule dynamics and the neurodegenerative triad of Alzheimer\u2019s disease: The hidden connection","volume":"143","author":"Brandt","year":"2017","journal-title":"J Neurochem"},{"key":"10.1016\/j.jare.2021.07.001_b0080","doi-asserted-by":"crossref","first-page":"203","DOI":"10.1177\/1756285611404470","article-title":"Disease-modifying treatments for Alzheimer\u2019s disease","volume":"4","author":"Galimberti","year":"2011","journal-title":"Ther Adv NeurolDisord"},{"key":"10.1016\/j.jare.2021.07.001_b0085","doi-asserted-by":"crossref","first-page":"4154","DOI":"10.1172\/JCI72992","article-title":"Expanding the genetic editing tool kit: ZFNs, TALENs, and CRISPR-Cas9","volume":"124","author":"Gupta","year":"2014","journal-title":"J Clin Invest"},{"key":"10.1016\/j.jare.2021.07.001_b0090","doi-asserted-by":"crossref","first-page":"5429","DOI":"10.1128\/jb.169.12.5429-5433.1987","article-title":"Nucleotide sequence of the iap gene, responsible for alkaline phosphatase isozyme conversion in Escherichia coli, and identification of the gene product","volume":"169","author":"IshinoY","year":"1987","journal-title":"J Bacteriol"},{"key":"10.1016\/j.jare.2021.07.001_b0095","doi-asserted-by":"crossref","first-page":"816","DOI":"10.1126\/science.1225829","article-title":"A programmable dual-RNA\u2013guided DNA endonuclease in adaptive bacterial immunity","volume":"337","author":"JinekM","year":"2012","journal-title":"Science"},{"key":"10.1016\/j.jare.2021.07.001_b0100","doi-asserted-by":"crossref","first-page":"1380","DOI":"10.1016\/j.cell.2013.08.021","article-title":"Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity","volume":"154","author":"Ran","year":"2013","journal-title":"Cell"},{"key":"10.1016\/j.jare.2021.07.001_b0105","doi-asserted-by":"crossref","first-page":"357","DOI":"10.1021\/acschembio.7b00855","article-title":"Type II-C CRISPR-Cas9 biology, mechanism, and application","volume":"13","author":"Mir","year":"2018","journal-title":"ACS Chem Biol"},{"key":"10.1016\/j.jare.2021.07.001_b0110","article-title":"Non-homologous end joining and homology directed DNA repair frequency of double-stranded breaks introduced by genome editing reagents","volume":"12","author":"ZaboikinM","year":"2017","journal-title":"PLoS ONE"},{"key":"10.1016\/j.jare.2021.07.001_b0115","doi-asserted-by":"crossref","unstructured":"TozzoP,ZulloS,CaenazzoL. Science Runs and the Debate Brakes: Somatic Gene-Editing as a New Tool for Gender-Specific Medicine in Alzheimer\u2019s Disease. Brain Sci. 2020;10:421.","DOI":"10.3390\/brainsci10070421"},{"issue":"3","key":"10.1016\/j.jare.2021.07.001_b0120","doi-asserted-by":"crossref","first-page":"458","DOI":"10.1038\/mt.2015.151","article-title":"Genome engineering using adeno-associated virus: basic and clinical research applications","volume":"24","author":"Gaj","year":"2016","journal-title":"Mol Ther"},{"issue":"4","key":"10.1016\/j.jare.2021.07.001_b0125","doi-asserted-by":"crossref","first-page":"660","DOI":"10.1016\/j.ymthe.2004.07.003","article-title":"Site-specific integration of functional transgenes into the human genome by adeno\/AAV hybrid vectors","volume":"10","author":"Recchia","year":"2004","journal-title":"Mol Ther"},{"issue":"4","key":"10.1016\/j.jare.2021.07.001_b0130","doi-asserted-by":"crossref","first-page":"281","DOI":"10.2174\/1566523034578285","article-title":"From virus evolution to vector revolution: use of naturally occurring serotypes of adeno-associated virus (AAV) as novel vectors for human gene therapy","volume":"3","author":"Grimm","year":"2003","journal-title":"Curr Gene Ther"},{"key":"10.1016\/j.jare.2021.07.001_b0135","doi-asserted-by":"crossref","unstructured":"Dissen GA, McBride J, Lomniczi A, et al. Using lentiviral vectors as delivery vehicles for gene therapy. Controlled genetic manipulations. Berlin: Springer; 2012. p. 69\u201396.","DOI":"10.1007\/978-1-61779-533-6_4"},{"key":"10.1016\/j.jare.2021.07.001_b0140","doi-asserted-by":"crossref","unstructured":"Sun J, Carlson-Stevermer J, Das U, et al. CRISPR\/Cas9 editing of APP C-terminus attenuates b-cleavage and promotes a-cleavage. Nat Commun. 2019;10(1):1\u20131.","DOI":"10.1038\/s41467-018-07971-8"},{"issue":"5","key":"10.1016\/j.jare.2021.07.001_b0145","first-page":"S94","article-title":"Caspase-6 knock-out using CRISPR\/Cas9 improves cognitive behavior in the 3xTg mouse model of Alzheimer\u2019s disease","volume":"20","author":"Offen","year":"2018","journal-title":"Cytotherapy"},{"issue":"5","key":"10.1016\/j.jare.2021.07.001_b0150","doi-asserted-by":"crossref","first-page":"S18","DOI":"10.1016\/j.jcyt.2018.02.036","article-title":"Towards gene-editing treatment for Alzheimer\u2019s disease: ApoE4 allele-specific knockout using a CRISPR cas9 variant","volume":"20","author":"Offen","year":"2018","journal-title":"Cytotherapy"},{"key":"10.1016\/j.jare.2021.07.001_b0155","doi-asserted-by":"crossref","first-page":"92","DOI":"10.1016\/S1474-4422(12)70259-4","article-title":"Genetic insights in alzheimer\u2019s disease","volume":"12","author":"Bettens","year":"2013","journal-title":"Lancet Neurol"},{"key":"10.1016\/j.jare.2021.07.001_b0160","doi-asserted-by":"crossref","first-page":"668","DOI":"10.1126\/science.1411576","article-title":"Genetic linkage evidence for a familial Alzheimer\u2019s disease locus on chromosome 14","volume":"258","author":"Schellenberg","year":"1992","journal-title":"Science"},{"key":"10.1016\/j.jare.2021.07.001_b0165","doi-asserted-by":"crossref","first-page":"970","DOI":"10.1126\/science.7638621","article-title":"A familial Alzheimer\u2019s disease locus on chromosome 1","volume":"269","author":"Levy-Lahad","year":"1995","journal-title":"Science"},{"key":"10.1016\/j.jare.2021.07.001_b0170","doi-asserted-by":"crossref","first-page":"4","DOI":"10.1186\/1750-1326-1-4","article-title":"Pathological and physiological functions of presenilins","volume":"1","author":"Vetrivel","year":"2006","journal-title":"Mol Neurodegener"},{"key":"10.1016\/j.jare.2021.07.001_b0175","doi-asserted-by":"crossref","first-page":"77","DOI":"10.1186\/s40478-017-0475-z","article-title":"CRISPR\/Cas9-correctable mutation-related molecular and physiological phenotypes in iPSC-derived Alzheimer\u2019s PSEN2 (N141I) neurons","volume":"5","author":"Ortiz-Virumbrales","year":"2017","journal-title":"Acta NeuropatholCommun"},{"key":"10.1016\/j.jare.2021.07.001_b0180","doi-asserted-by":"crossref","first-page":"285","DOI":"10.1016\/j.scr.2016.08.002","article-title":"Generation of a gene-corrected isogenic control cell line from an Alzheimer\u2019s disease patient iPSC line carrying a A79V mutation in PSEN1","volume":"17","author":"Pires","year":"2016","journal-title":"Stem Cell Res"},{"key":"10.1016\/j.jare.2021.07.001_b0185","doi-asserted-by":"crossref","first-page":"466","DOI":"10.1016\/j.scr.2016.09.018","article-title":"Generation of a gene-corrected isogenic control hiPSC line derived from a familial Alzheimer\u2019s disease patient carrying a L150P mutation in presenilin 1","volume":"17","author":"Poon","year":"2016","journal-title":"Stem Cell Res"},{"key":"10.1016\/j.jare.2021.07.001_b0190","doi-asserted-by":"crossref","first-page":"429","DOI":"10.1016\/j.omtn.2018.03.007","article-title":"CRISPR\/Cas9 mediated disruption of the swedish APP allele as a therapeutic approach for early-onset alzheimer\u2019s disease","volume":"11","author":"Gy\u00f6rgy","year":"2018","journal-title":"Mol Ther Nucleic Acids"},{"key":"10.1016\/j.jare.2021.07.001_b0195","doi-asserted-by":"crossref","first-page":"345","DOI":"10.1038\/ng0892-345","article-title":"A pathogenic mutation for probable Alzheimer\u2019s disease in the APP gene at the N-terminus of beta-amyloid","volume":"1","author":"Mullan","year":"1992","journal-title":"Nat Genet"},{"key":"10.1016\/j.jare.2021.07.001_b0200","doi-asserted-by":"crossref","first-page":"447","DOI":"10.1016\/j.neuron.2013.05.035","article-title":"Activity-induced convergence of APP and BACE-1 in acidic microdomains via an endocytosis-dependent pathway","volume":"79","author":"Das","year":"2013","journal-title":"Neuron"},{"key":"10.1016\/j.jare.2021.07.001_b0205","doi-asserted-by":"crossref","first-page":"S20","DOI":"10.1038\/475S20a","article-title":"Finding risk factors","volume":"475","author":"Genetics","year":"2011","journal-title":"Nature"},{"key":"10.1016\/j.jare.2021.07.001_b0210","doi-asserted-by":"crossref","first-page":"9077","DOI":"10.1016\/S0021-9258(19)52510-8","article-title":"Human E apoprotein heterogeneity. Cysteine-arginine interchanges in the amino acid sequence of the apo-E isoforms","volume":"256","author":"Weisgraber","year":"1981","journal-title":"J Biol Chem"},{"key":"10.1016\/j.jare.2021.07.001_b0215","doi-asserted-by":"crossref","first-page":"1349","DOI":"10.1001\/jama.1997.03550160069041","article-title":"Effects of age, sex, and ethnicity on the association between apolipoprotein E genotype and Alzheimer disease. A meta-analysis. APOE and alzheimer disease meta-analysis consortium","volume":"278","author":"Farrer","year":"1997","journal-title":"JAMA"},{"key":"10.1016\/j.jare.2021.07.001_b0220","doi-asserted-by":"crossref","first-page":"1467","DOI":"10.1212\/WNL.43.8.1467","article-title":"Association of apolipoprotein E allele epsilon 4 with late-onset familial and sporadic Alzheimer\u2019s disease","volume":"43","author":"Saunders","year":"1993","journal-title":"Neurol"},{"key":"10.1016\/j.jare.2021.07.001_b0225","doi-asserted-by":"crossref","first-page":"740","DOI":"10.1016\/j.neuron.2014.01.045","article-title":"ApoE and A\u03b2 in Alzheimer\u2019s disease: accidental encounters or partners?","volume":"81","author":"Kanekiyo","year":"2014","journal-title":"Neuron"},{"key":"10.1016\/j.jare.2021.07.001_b0230","doi-asserted-by":"crossref","first-page":"647","DOI":"10.1038\/s41591-018-0004-z","article-title":"Gain of toxic apolipoprotein E4 effects in human iPSC-derived neurons is ameliorated by a small-molecule structure corrector","volume":"24","author":"Wang","year":"2018","journal-title":"Nat Med"},{"key":"10.1016\/j.jare.2021.07.001_b0235","doi-asserted-by":"crossref","first-page":"19053","DOI":"10.1074\/jbc.271.32.19053","article-title":"Human apolipoprotein E4 domain interaction. Arginine 61 and glutamic acid 255 interact to direct the preference for very low density lipoproteins","volume":"271","author":"Dong","year":"1996","journal-title":"J Biol Chem"},{"issue":"1","key":"10.1016\/j.jare.2021.07.001_b0240","doi-asserted-by":"crossref","first-page":"33","DOI":"10.1186\/s13024-018-0265-5","article-title":"iPSCderived familial Alzheimer\u2019s PSEN2 N141I cholinergic neurons exhibit mutation-dependent molecular pathology corrected by insulin signaling","volume":"13","author":"Moreno","year":"2018","journal-title":"Mol Neurodegener"},{"issue":"4","key":"10.1016\/j.jare.2021.07.001_b0245","doi-asserted-by":"crossref","first-page":"524","DOI":"10.1038\/s41593-019-0352-0","article-title":"In vivo neuronal gene editing via CRISPR-Cas9 amphiphilic nanocomplexes alleviates deficits in mouse models of Alzheimer\u2019sdisease","volume":"22","author":"Park","year":"2019","journal-title":"Nat Neurosci"},{"issue":"2","key":"10.1016\/j.jare.2021.07.001_b0250","doi-asserted-by":"crossref","first-page":"571","DOI":"10.3233\/JAD-171058","article-title":"Generation of a new tau knockout (tau Dex1) line using CRISPR\/Cas9 genome editing in mice","volume":"62","author":"Tan","year":"2018","journal-title":"J Alzheimers Dis"},{"issue":"7601","key":"10.1016\/j.jare.2021.07.001_b0255","doi-asserted-by":"crossref","first-page":"125","DOI":"10.1038\/nature17664","article-title":"Efficient introductionof specific homozygous and heterozygousmutations using CRISPR\/Cas9","volume":"533","author":"Paquet","year":"2016","journal-title":"Nature"},{"issue":"1\u20132","key":"10.1016\/j.jare.2021.07.001_b0260","doi-asserted-by":"crossref","first-page":"33","DOI":"10.1016\/j.neulet.2006.06.072","article-title":"Chinese presenilin-1 V97Lmutation enhanced Ab42 levels in SH-SY5Y neuroblastomacells","volume":"406","author":"Fang","year":"2006","journal-title":"Neurosci Lett"},{"issue":"7603","key":"10.1016\/j.jare.2021.07.001_b0265","doi-asserted-by":"crossref","first-page":"420","DOI":"10.1038\/nature17946","article-title":"Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage","volume":"533","author":"Komor","year":"2016","journal-title":"Nature"},{"issue":"1","key":"10.1016\/j.jare.2021.07.001_b0270","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1038\/celldisc.2016.26","article-title":"Alzheimer\u2019s disease-associated mutations increaseamyloid precursor protein resistance to c-secretasecleavage and the Ab42\/Ab40 ratio","volume":"2","author":"Xu","year":"2016","journal-title":"Cell Discov"},{"key":"10.1016\/j.jare.2021.07.001_b0275","doi-asserted-by":"crossref","unstructured":"Sud hanshu P. Raikwar, Ramasamy Thangavel, Iuliia Dubova, Govindhasamy Pushpavathi Selvakumar, Mohammad Ejaz Ahmed, Duraisamy Kempuraj, Smita A. Zaheer, Shankar S. Iyer, Asgar Zaheer, Targeted gene editing of glia maturation factor in microglia: a novel Alzheimer\u2019s disease therapeutic target, Mol. Neurobiol. 56 (2019) 378\u2013393.","DOI":"10.1007\/s12035-018-1068-y"},{"key":"10.1016\/j.jare.2021.07.001_b0280","doi-asserted-by":"crossref","first-page":"1800","DOI":"10.1038\/s41467-018-04238-0","article-title":"Generation of App knock-in mice reveals deletion mutations protective against Alzheimer\u2019s diseaselike pathology","volume":"9","author":"Nagata","year":"2018","journal-title":"Nat Commun"},{"key":"10.1016\/j.jare.2021.07.001_b0285","doi-asserted-by":"crossref","first-page":"6385","DOI":"10.1073\/pnas.1820160116","article-title":"GSAP modulates \u03b3-secretase specificity by inducing conformational change in PS1","volume":"116","author":"Wong","year":"2019","journal-title":"Proc Natl Acad Sci"},{"key":"10.1016\/j.jare.2021.07.001_b0290","doi-asserted-by":"crossref","first-page":"3","DOI":"10.1186\/1756-6606-4-3","article-title":"APP processing in Alzheimer\u2019s disease","volume":"4","author":"Zhang","year":"2011","journal-title":"Mol Brain"},{"key":"10.1016\/j.jare.2021.07.001_b0295","doi-asserted-by":"crossref","first-page":"726","DOI":"10.1002\/ana.25455","article-title":"Neuronal apolipoprotein E4 increases cell death and phosphorylated tau release in alzheimer disease","volume":"85","author":"Wadhwani","year":"2019","journal-title":"Ann Neurol"},{"key":"10.1016\/j.jare.2021.07.001_b0300","doi-asserted-by":"crossref","first-page":"12029","DOI":"10.1002\/anie.201506030","article-title":"Self-assembled DNA nanoclews for the efficient delivery of CRISPR-Cas9 for genome editing","volume":"54","author":"Sun","year":"2015","journal-title":"Angew Chem Int Ed"},{"key":"10.1016\/j.jare.2021.07.001_b0305","article-title":"Nanoparticles-mediated CRISPR\/Cas9 delivery: recent advances in cancer treatment","volume":"56","author":"Aghamiri","year":"2020","journal-title":"J Drug Deliv Sci Technol"},{"issue":"3","key":"10.1016\/j.jare.2021.07.001_b0310","doi-asserted-by":"crossref","first-page":"146","DOI":"10.1089\/nat.2018.0721","article-title":"Lipid nanoparticles enabling gene therapies: from concepts to clinical utility","volume":"28","author":"Kulkarni","year":"2018","journal-title":"Nucleic Acid Ther"},{"issue":"1","key":"10.1016\/j.jare.2021.07.001_b0315","doi-asserted-by":"crossref","first-page":"136","DOI":"10.1016\/j.cell.2020.03.023","article-title":"CRISPR-based therapeutic genome editing: strategies and in vivo delivery byAAV vectors","volume":"181","author":"Wang","year":"2020","journal-title":"Cell"},{"key":"10.1016\/j.jare.2021.07.001_b0320","doi-asserted-by":"crossref","first-page":"149","DOI":"10.1038\/s41586-019-1711-4","article-title":"Search-and-replace genome editing without double-strand breaks or donor DNA","volume":"576","author":"Anzalone","year":"2019","journal-title":"Nature"},{"key":"10.1016\/j.jare.2021.07.001_b0325","doi-asserted-by":"crossref","first-page":"27","DOI":"10.1016\/j.stem.2014.04.020","article-title":"Low incidence of off-target mutations in individual CRISPR-Cas9 and TALEN targeted human stem cell clones detected by whole-genome sequencing","volume":"15","author":"Veres","year":"2014","journal-title":"Cell Stem Cell"},{"key":"10.1016\/j.jare.2021.07.001_b0330","doi-asserted-by":"crossref","first-page":"95","DOI":"10.1038\/nature17939","article-title":"Parkinson-associated risk variant in distal enhancer of \u03b1-synuclein modulates target gene expression","volume":"533","author":"Soldner","year":"2016","journal-title":"Nature"},{"key":"10.1016\/j.jare.2021.07.001_b0335","doi-asserted-by":"crossref","first-page":"1423","DOI":"10.1016\/j.stemcr.2017.08.026","article-title":"FACS-assisted CRISPR-Cas9 genome editing facilitates Parkinson\u2019s disease modeling","volume":"9","author":"Arias-Fuenzalida","year":"2017","journal-title":"Stem Cell Rep"},{"key":"10.1016\/j.jare.2021.07.001_b0340","doi-asserted-by":"crossref","first-page":"44","DOI":"10.1016\/j.scr.2017.08.013","article-title":"CRISPR\/cas9 and piggyBac-mediated footprintfree LRRK2-G2019S knock-in reveals neuronal complexity phenotypes and \u03b1Synuclein modulation in dopaminergic neurons","volume":"24","author":"Qing","year":"2017","journal-title":"Stem Cell Res"},{"key":"10.1016\/j.jare.2021.07.001_b0345","doi-asserted-by":"crossref","first-page":"E180","DOI":"10.1073\/pnas.1711023115","article-title":"Genome-wide CRISPR screen for PARKIN regulators reveals transcriptional repression as a determinant of mitophagy","volume":"115","author":"Potting","year":"2018","journal-title":"Proc Natl Acad Sci U S A"},{"key":"10.1016\/j.jare.2021.07.001_b0350","doi-asserted-by":"crossref","first-page":"333","DOI":"10.1093\/toxsci\/kfz049","article-title":"Mechanistic interplay between autophagy and apoptotic signaling in Endosulfaninduced dopaminergic neurotoxicity: relevance to the adverse outcome pathway in pesticide neurotoxicity","volume":"169","author":"Song","year":"2019","journal-title":"Toxicol Sci"},{"key":"10.1016\/j.jare.2021.07.001_b0355","doi-asserted-by":"crossref","first-page":"12932","DOI":"10.1038\/ncomms12932","article-title":"Prokineticin-2 upregulation during neuronal injury mediates a compensatory protective response against dopaminergic neuronal degeneration","volume":"7","author":"Gordon","year":"2016","journal-title":"Nat Commun"},{"key":"10.1016\/j.jare.2021.07.001_b0360","doi-asserted-by":"crossref","first-page":"643","DOI":"10.1007\/s11481-013-9439-7","article-title":"Glia maturation factor induces interleukin-33 release from astrocytes: implications for neurodegenerative diseases","volume":"8","author":"Kempuraj","year":"2013","journal-title":"J NeuroImmune Pharmacol"},{"key":"10.1016\/j.jare.2021.07.001_b0365","first-page":"1","article-title":"CRISPR\/Cas9 editing of glia maturation factor regulates mitochondrial dynamics by attenuation of the NRF2\/HO-1 dependent ferritin activation in glial cells","author":"Selvakumar","year":"2019","journal-title":"J NeuroImmune Pharmacol"},{"key":"10.1016\/j.jare.2021.07.001_b0370","doi-asserted-by":"crossref","first-page":"30","DOI":"10.3389\/fnmol.2016.00030","article-title":"CRISPR\/Cas9: implications for modeling and therapy of neurodegenerative diseases","volume":"9","author":"Yang","year":"2016","journal-title":"Front Mol Neurosci"},{"key":"10.1016\/j.jare.2021.07.001_b0375","doi-asserted-by":"crossref","first-page":"510","DOI":"10.1111\/ejn.14286","article-title":"Engineering synucleinopathy-resistant human dopaminergic neurons by CRISPR-mediated deletion of the SNCA gene","volume":"49","author":"Chen","year":"2019","journal-title":"Eur J Neurosci"},{"key":"10.1016\/j.jare.2021.07.001_b0380","doi-asserted-by":"crossref","first-page":"524","DOI":"10.1038\/s41593-019-0352-0","article-title":"In vivo neuronal gene editing via CRISPR\u2013Cas9 amphiphilic nanocomplexes alleviates deficits in mouse models of Alzheimer\u2019s disease","volume":"22","author":"Park","year":"2019","journal-title":"Nat Neurosci"},{"key":"10.1016\/j.jare.2021.07.001_b0385","doi-asserted-by":"crossref","first-page":"12","DOI":"10.1016\/j.ymthe.2016.11.010","article-title":"CRISPR\/Cas9 editing of the mutant huntingtin allele in vitro and in vivo","volume":"25","author":"Monteys","year":"2017","journal-title":"Mol Ther"},{"key":"10.1016\/j.jare.2021.07.001_b0390","doi-asserted-by":"crossref","first-page":"227","DOI":"10.1016\/S0896-6273(03)00366-0","article-title":"Huntingtin and huntingtin-associated protein 1 influence neuronal calcium signaling mediated by inositol-(1, 4, 5) triphosphate receptor type 1","volume":"39","author":"Tang","year":"2003","journal-title":"Neuron"},{"key":"10.1016\/j.jare.2021.07.001_b0395","doi-asserted-by":"crossref","first-page":"35","DOI":"10.3233\/JHD-170266","article-title":"Inhibition of TRPC1-dependent store-operated calcium entry improves synaptic stability and motor performance in a mouse model of Huntington\u2019s disease","volume":"7","author":"Wu","year":"2018","journal-title":"J Huntingt Dis"},{"key":"10.1016\/j.jare.2021.07.001_b0400","doi-asserted-by":"crossref","first-page":"5110","DOI":"10.1093\/nar\/gkf664","article-title":"An upstream open reading frame impedes translation of the huntingtin gene","volume":"30","author":"Lee","year":"2002","journal-title":"Nucleic Acids Res"},{"key":"10.1016\/j.jare.2021.07.001_b0405","doi-asserted-by":"crossref","first-page":"754","DOI":"10.3390\/ijms18040754","article-title":"CRISPRCas9 mediated gene-silencing of the mutant huntingtin gene in an in vitro model of Huntington\u2019s disease","volume":"18","author":"Kolli","year":"2017","journal-title":"Int J Mol Sci"},{"key":"10.1016\/j.jare.2021.07.001_b0410","doi-asserted-by":"crossref","unstructured":"Kantor B, Tagliafierro L, Gu J, Zamora ME, Ilich E, Grenier C, Huang ZY, Murphy S, Chiba-Falek O. Downregulation of SNCA Expression by Targeted Editing of DNA Methylation: A Potential Strategy for Precision Therapy in PD. Mol Ther. 2018 Nov 7;26(11):2638-2649. doi: 10.1016\/j.ymthe.2018.08.019. Epub 2018 Aug 29. PMID: 30266652; PMCID: PMC6224806.","DOI":"10.1016\/j.ymthe.2018.08.019"},{"key":"10.1016\/j.jare.2021.07.001_b0415","doi-asserted-by":"crossref","first-page":"1175","DOI":"10.1007\/s00018-014-1744-7","article-title":"Generation of CRISPR\/Cas9-mediated gene-targeted pigs via somatic cell nuclear transfer","volume":"72","author":"Zhou","year":"2015","journal-title":"Cell Mol Life Sci"},{"key":"10.1016\/j.jare.2021.07.001_b0420","doi-asserted-by":"crossref","first-page":"1423","DOI":"10.1016\/j.stemcr.2017.08.026","article-title":"Sarah Louise Nickels, Holm Zaehres, Hans Robert Sch\u00f6ler, Jens Christian Schwamborn, FACS-assisted CRISPR-Cas9 genome editing facilitates Parkinson\u2019s disease modeling","volume":"9","author":"Arias-Fuenzalida","year":"2017","journal-title":"Stem Cell Rep"},{"key":"10.1016\/j.jare.2021.07.001_b0425","doi-asserted-by":"crossref","first-page":"20620","DOI":"10.1038\/srep20620","article-title":"One-step generation of triple gene-targeted pigs using CRISPR\/Cas9 system","volume":"6","author":"Wang","year":"2016","journal-title":"Sci Rep"},{"key":"10.1016\/j.jare.2021.07.001_b0430","doi-asserted-by":"crossref","first-page":"2719","DOI":"10.1172\/JCI92087","article-title":"Ha Eun Kong, Xiaobo Sun, Zhaohui Qin, Peng Jin, Shihua Li, CRISPR\/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of Huntington\u2019s disease","volume":"127","author":"Yang","year":"2017","journal-title":"J Clin Invest"},{"key":"10.1016\/j.jare.2021.07.001_b0435","doi-asserted-by":"crossref","unstructured":"Jun Wan Shin, Kyung-Hee Kim, Michael J. Chao, Ranjit S. Atwal, Tammy Gillis, Marcy E. MacDonald, James F. Gusella, Jong-Min Lee, Permanent inactivation of Huntington\u2019s disease mutation by personalized allele-specific CRISPR\/Cas9, Hum.Mol. Genet. 25(2016) 4566-4576.","DOI":"10.1093\/hmg\/ddw286"},{"key":"10.1016\/j.jare.2021.07.001_b0440","doi-asserted-by":"crossref","first-page":"35","DOI":"10.3233\/JHD-170266","article-title":"Inhibition of TRPC1-dependent store-operated calcium entry improves synaptic stability and motor performance in a mouse model of Huntington\u2019s disease","volume":"7","author":"Jun","year":"2018","journal-title":"J Huntingt Dis"},{"key":"10.1016\/j.jare.2021.07.001_b0445","doi-asserted-by":"crossref","first-page":"754","DOI":"10.3390\/ijms18040754","article-title":"CRISPRCas9 mediated gene-silencing of the mutant huntingtin gene in an in vitro model of Huntington\u2019s disease","volume":"18","author":"Nivya Kolli","year":"2017","journal-title":"Int J Mol Sci"},{"key":"10.1016\/j.jare.2021.07.001_b0450","doi-asserted-by":"crossref","first-page":"23","DOI":"10.1016\/j.ddtec.2018.05.001","article-title":"CRISPR\/Cas9 genome surgery for retinal diseases","volume":"28","author":"Xu","year":"2018","journal-title":"Drug Discov Today Technol"},{"issue":"3","key":"10.1016\/j.jare.2021.07.001_b0455","doi-asserted-by":"crossref","first-page":"13","DOI":"10.1167\/tvst.6.3.13","article-title":"CRISPR-Cas Genome Surgery in Ophthalmology","volume":"6","author":"DiCarlo","year":"2017","journal-title":"Transl Vis Sci Technol"},{"key":"10.1016\/j.jare.2021.07.001_b0460","doi-asserted-by":"crossref","DOI":"10.1016\/j.lfs.2020.118165","article-title":"CRISPR\/Cas9 novel therapeutic road for the treatment of neurodegenerative diseases","volume":"259","author":"Karimian","year":"2020","journal-title":"Life Sci"},{"issue":"13","key":"10.1016\/j.jare.2021.07.001_b0465","doi-asserted-by":"crossref","first-page":"2283","DOI":"10.7150\/ijbs.33481","article-title":"CRISPR\/Cas9-related technologies in liver diseases: from feasibility to future diversity","volume":"16","author":"Xu","year":"2020","journal-title":"Int J Biol Sci"},{"issue":"1","key":"10.1016\/j.jare.2021.07.001_b0470","doi-asserted-by":"crossref","first-page":"43","DOI":"10.1186\/s12916-017-0798-4","article-title":"Genome editing for inborn errors of metabolism: advancing towards the clinic","volume":"15","author":"Schneller","year":"2017","journal-title":"BMC Med"},{"issue":"1","key":"10.1016\/j.jare.2021.07.001_b0475","doi-asserted-by":"crossref","first-page":"122","DOI":"10.1016\/j.jacbts.2018.11.004","article-title":"Therapeutic Genome Editing in Cardiovascular Diseases","volume":"4","author":"German","year":"2019","journal-title":"JACC Basic Transl Sci"},{"key":"10.1016\/j.jare.2021.07.001_b0480","doi-asserted-by":"crossref","first-page":"13","DOI":"10.1016\/j.semcdb.2019.05.026","article-title":"Advancements in CRISPR\/Cas9 technology-Focusing on cancer therapeutics and beyond","volume":"96","author":"Mirza","year":"2019","journal-title":"Semin Cell Dev Biol"},{"key":"10.1016\/j.jare.2021.07.001_b0485","doi-asserted-by":"crossref","first-page":"2151","DOI":"10.1021\/mp200100f","article-title":"Targeted delivery of cargoes into a murine solid tumor by a cell-penetrating peptide and cleavable poly (ethylene glycol) comodified liposomal delivery system via systemic administration","volume":"8","author":"Kuai","year":"2011","journal-title":"Mol Pharm"},{"key":"10.1016\/j.jare.2021.07.001_b0490","doi-asserted-by":"crossref","first-page":"309","DOI":"10.1016\/S1474-4422(11)70039-4","article-title":"AAV2-GAD gene therapy for advanced Parkinson\u2019s disease: A double-blind, sham-surgery controlled, randomised trial","volume":"10","author":"LeWitt","year":"2011","journal-title":"Lancet Neurol"}],"container-title":["Journal of Advanced Research"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2090123221001351?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2090123221001351?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,10,30]],"date-time":"2025-10-30T20:52:16Z","timestamp":1761857536000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2090123221001351"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,9]]},"references-count":98,"alternative-id":["S2090123221001351"],"URL":"https:\/\/doi.org\/10.1016\/j.jare.2021.07.001","relation":{},"ISSN":["2090-1232"],"issn-type":[{"value":"2090-1232","type":"print"}],"subject":[],"published":{"date-parts":[[2022,9]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"CRISPR\/Cas9 gene editing: New hope for Alzheimer's disease therapeutics","name":"articletitle","label":"Article Title"},{"value":"Journal of Advanced Research","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.jare.2021.07.001","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2022 The Authors. Published by Elsevier B.V. on behalf of Cairo University.","name":"copyright","label":"Copyright"}]}}