Vigour of CRISPR/Cas9 Gene Editing in Alzheimer’s Disease
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Abstract
Ailment repairing regiments has turn out to be arduous, despite a plenty of understanding and knowledge acquired in the past relating to the molecular underpinnings of Alzheimer’s disease (AD. Umpteen clinical experiments targeting the fabrication and accumulation have been turned fruitless to fit potency standards. The tests aiming beta-amyloid hypothesis also turned futile making it exigent for further handling tactics. The new emanation of a comparably candid, economical, and punctilious system known as gene editing have showed light in path of cure for AD by CRISPR/Cas9 gene editing. Being a straight approach this procedure has already shown assurance in other neurological disorders too such as Huntington’s disease. This review standpoint the immanent service of CRISPR/Cas9 as a remedial option for AD by aiming on specific genes inclusive of those that induce early-onset AD, as well as those that are substantial risk components for late-onset AD such as the apolipoprotein E4 (APOE4) gene.
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Copyright (c) 2018 Paul J.
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Jones EL, Kalaria RN, Sharp SI, O’Brien JT, Francis PT, et al. Genetic associations of autopsy-confirmed vascular dementia subtypes. Dement GeriatrCognDisord. 2011; 31: 247–253. Ref.: https://goo.gl/VrBuco
Mirra SS, Heyman A, McKeel D, Sumi SM, Crain BJ, et al. The consortium to establish a registry for Alzheimer’s disease (CERAD) part II. Standardization of the neuropathological assessment of Alzheimer’s disease. Neurol. 1991; 41: 479–486. Ref.: https://goo.gl/GZ4PKL
Terry RD, Masliah E, Salmon DP, Butters N, DeTeresa R, et al. Physical basis of cognitive alterations in Alzheimer’s disease: synapse loss is the major correlate of cognitive impairment. Ann Neurol. 1991; 30: 572–580. Ref.: https://goo.gl/UBTCiK
DeKosky ST, Scheff SW, Styren SD. Structural correlates of cognition in dementia: Quantification and assessment of synapse change. Neurodegeneration. 1996; 5: 417–421. Ref.: https://goo.gl/vxUsux
Coleman PD, Yao PJ. Synaptic slaughter in Alzheimer’s disease. Neurobiol Aging. 2003; 24: 1023–1027. Ref.: https://goo.gl/nezMzY
Lacor PN, Buniel MC, Chang L, Fernandez SJ, Gong Y, et al. Synaptic targeting by Alzheimer’s-related amyloid beta oligomers. J Neurosci. 2004; 24: 10191–10200. Ref.: https://goo.gl/st1b1F
Lambert MP, Barlow AK, Chromy BA, Edwards C, Freed R, et al. Diffusible, nonfibrillar ligands derived from Abeta1-42 are potent central nervous system neurotoxins. Proc Natl Acad Sci U S A. 1998; 95: 6448–6453. Ref.: https://goo.gl/wyxW1e
McLean CA, Cherny RA, Fraser FW, Fuller SJ, Smith MJ, et al. Soluble pool of Abeta amyloid as a determinant of severity of neurodegeneration in Alzheimer’s disease. Ann Neurol. 1999; 46: 860–866. Ref.: https://goo.gl/ETDLih
Broersen K, Rousseau F, Schymkowitz J. The culprit behind amyloid beta peptide related neurotoxicity in Alzheimer’s disease: Oligomer size or conformation? Alzheimers Res Ther. 2010; 2: 12. Ref.: https://goo.gl/sSVGiS
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: 439. Ref.: https://goo.gl/7NDf8r
Mojica FJ, Diez-Villasenor C, Garcia-Martinez J, Soria E. Intervening sequences of regularly spaced prokaryotic repeats derive from foreign genetic elements. J MolEvol. 2005; 60: 174–182. Ref.: https://goo.gl/oKLqZy
Pourcel C, Salvignol G, Vergnaud G. CRISPR elements in Yersinia pestis acquire new repeats by preferential uptake of bacteriophage DNA, and provide additional tools for evolutionary studies. Microbiology. 2005; 151: 653–663. Ref.: https://goo.gl/wpw3tY
Barrangou R, Fremaux C, Deveau H, Richards M, Boyaval P, et al. CRISPR provides acquired resistance against viruses in prokaryotes. Science. 2007; 315: 1709–1712. Ref.: https://goo.gl/jRYzbU
Bolotin A, Quinquis B, Sorokin A, Ehrlich SD. Clustered regularly interspaced short palindrome repeats (CRISPRs) have spacers of extrachromosomal origin. Microbiology. 2005; 151: 2551–2561. Ref.: https://goo.gl/j9tdyj
Deltcheva E, Chylinski K, Sharma CM, Gonzales K, Chao Y, et al. CRISPR RNA maturation by trans-encoded small RNA and host factor RNase III. Nature. 2011; 471: 602–607. Ref.: https://goo.gl/LgTULX
Saudou F, Humbert S. The biology of huntingtin. Neuron. 2016; 89: 910–926. Ref.: https://goo.gl/WGjZZv
Bates GP, Dorsey R, Gusella JF, Hayden MR, Kay C, et al. Huntington disease. Nat Rev Dis Primers. 2015; 1: 15005. Ref.: https://goo.gl/sYTJU7
Dabrowska M, Juzwa W, Krzyzosiak WJ, Olejniczak M. Precise excision of the CAG tract from the huntingtin gene by cas9 nickases. Front Neurosci. 2018; 12: 75. Ref.: https://goo.gl/juHxQe
Schellenberg GD, Bird TD, Wijsman EM, Orr HT, Anderson L, et al. Genetic linkage evidence for a familial Alzheimer’s disease locus on chromosome 14. Science. 1992; 258: 668–671. Ref.: https://goo.gl/q2EmRY
Levy-Lahad E, Wijsman EM, Nemens E, Anderson L, Goddard KA, et al. A familial Alzheimer’s disease locus on chromosome 1. Science. 1995; 269: 970–973. Ref.: https://goo.gl/2SYDSQ
Vetrivel KS, Zhang YW, Xu H, Thinakaran G. Pathological and physiological functions of presenilins. MolNeurodegener. 2006; 1: 4. Ref.: https://goo.gl/Dbk6wU
Pires C, Schmid B, Petræus C, Poon A, Nimsanor N, et al. Generation of a gene-corrected isogenic control cell line from an Alzheimer’s disease patient iPSC line carrying a A79V mutation in PSEN1. Stem Cell Res. 2016; 17: 285–288. Ref.: https://goo.gl/Bns14i
Poon A, Schmid B, Pires C, Nielsen TT, Hjermind LE, et al. Generation of a gene-corrected isogenic control hiPSC line derived from a familial Alzheimer’s disease patient carrying a L150P mutation in presenilin 1. Stem Cell Res. 2016; 17: 466–469. Ref.: https://goo.gl/TAsC6J
György B, Lööv C, Zaborowski M, Takeda S, Kleinstiver B, et al. CRISPR/Cas9 mediated disruption of the swedish APP allele as a therapeutic approach for early-onset alzheimer’s disease. MolTher Nucleic Acids. 2018; 11: 429–440. Ref.: https://goo.gl/5Q2R3R
Sun J, Carlson-Stevermer J, Das U, Shen M, Delenclos M, et al. A CRISPR/Cas9 based strategy to manipulate the Alzheimer’s amyloid pathway. 2018 bioRxiv. Ref.: https://goo.gl/3NdTq9
Das U, Scott DA, Ganguly A, Koo EH, Tang Y, et al. Activity-induced convergence of APP and BACE-1 in acidic microdomains via an endocytosis-dependent pathway. Neuron. 2013; 79: 447–460. Ref.: https://goo.gl/Q3nKnq
Eisenstein M. Genetics: Finding risk factors. Nature. 2011; 475: S20–22. Ref.: https://goo.gl/GNjWRL
Weisgraber KH, Rall SC, Jr, Mahley RW. Human E apoprotein heterogeneity. Cysteine-arginine interchanges in the amino acid sequence of the apo-E isoforms. J Biol Chem. 1981; 256: 9077–9083. Ref.: https://goo.gl/dn7yof
Farrer LA, Cupples LA, Haines JL, Hyman B, Kukull WA, et al. 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. JAMA. 1997; 278: 1349–1356. Ref.: https://goo.gl/VTAWzn
Saunders AM, Strittmatter WJ, Schmechel D, George-Hyslop PH, Pericak-Vance MA, et al. Association of apolipoprotein E allele epsilon 4 with late-onset familial and sporadic Alzheimer’s disease. Neurol. 1993; 43: 1467–1472. Ref.: https://goo.gl/nTC61T
Kanekiyo T, Xu H, Bu G. ApoE and Aβ in Alzheimer’s disease: accidental encounters or partners? Neuron. 2014; 81: 740–754. Ref.: https://goo.gl/vuR6oA
Wang C, Najm R, Xu Q, Jeong DE, Walker D, et al. Gain of toxic apolipoprotein E4 effects in human iPSC-derived neurons is ameliorated by a small-molecule structure corrector. Nat Med. 2018; 24: 647–657. Ref.: https://goo.gl/rvcjWZ
Dong LM, Weisgraber KH. Human apolipoprotein E4 domain interaction. Arginine 61 and glutamic acid 255 interact to direct the preference for very low density lipoproteins. J Biol Chem. 1996; 271: 19053–19057. Ref.: https://goo.gl/bff8mX
Prince M, Comas-Herrera A, Knapp M, Guerchet M, Karagiannidou M. Coverage, quality and costs now and in the future. Alzheimer’s Disease International; London: 2016. World Alzheimer Report 2016. Improving healthcare for people living with dementia.