Delivery Platforms for CRISPR/Cas9 Genome Editing of Glial Cells in the Central Nervous System.
CRISPR/Cas9 system
adeno-associated viral vectors
astrocytes
editing
lentiviral vectors
microglia
nanoparticles
oligodendrocytes
Journal
Frontiers in genome editing
ISSN: 2673-3439
Titre abrégé: Front Genome Ed
Pays: Switzerland
ID NLM: 101775540
Informations de publication
Date de publication:
2021
2021
Historique:
received:
20
12
2020
accepted:
21
01
2021
entrez:
29
10
2021
pubmed:
30
10
2021
medline:
30
10
2021
Statut:
epublish
Résumé
Glial cells (astrocytes, oligodendrocytes, and microglia) are emerging as key players in several physiological and pathological processes of the central nervous system (CNS). Astrocytes and oligodendrocytes are not only supportive cells that release trophic factors or regulate energy metabolism, but they also actively modulate critical neuronal processes and functions in the tripartite synapse. Microglia are defined as CNS-resident cells that provide immune surveillance; however, they also actively contribute to shaping the neuronal microenvironment by scavenging cell debris or regulating synaptogenesis and pruning. Given the many interconnected processes coordinated by glial cells, it is not surprising that both acute and chronic CNS insults not only cause neuronal damage but also trigger complex multifaceted responses, including neuroinflammation, which can critically contribute to the disease progression and worsening of symptoms in several neurodegenerative diseases. Overall, this makes glial cells excellent candidates for targeted therapies to treat CNS disorders. In recent years, the application of gene editing technologies has redefined therapeutic strategies to treat genetic and age-related neurological diseases. In this review, we discuss the advantages and limitations of clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9-based gene editing in the treatment of neurodegenerative disorders, focusing on the development of viral- and nanoparticle-based delivery methods for
Identifiants
pubmed: 34713256
doi: 10.3389/fgeed.2021.644319
pmc: PMC8525379
doi:
Types de publication
Journal Article
Review
Langues
eng
Pagination
644319Informations de copyright
Copyright © 2021 Meneghini, Peviani, Luciani, Zambonini and Gritti.
Déclaration de conflit d'intérêts
The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.
Références
Genome Res. 2015 Aug;25(8):1158-69
pubmed: 26025803
Int J Dev Biol. 2010;54(11-12):1589-96
pubmed: 21404180
Exp Neurol. 2019 Sep;319:112813
pubmed: 30171864
Nat Rev Drug Discov. 2018 Sep;17(9):641-659
pubmed: 30093643
Acta Neuropathol Commun. 2016 Jul 11;4(1):69
pubmed: 27402089
Nat Methods. 2013 Oct;10(10):977-9
pubmed: 23892898
Int J Mol Sci. 2019 Jan 29;20(3):
pubmed: 30699952
Nat Commun. 2018 Jun 5;9(1):2184
pubmed: 29872041
J Exp Neurosci. 2016 Aug 08;10(Suppl 1):1-9
pubmed: 27547038
Nat Methods. 2013 Oct;10(10):973-6
pubmed: 23892895
Nat Nanotechnol. 2019 Oct;14(10):974-980
pubmed: 31501532
Neuron. 2017 Feb 8;93(3):574-586.e8
pubmed: 28111081
Nat Biotechnol. 2009 Jan;27(1):59-65
pubmed: 19098898
Biomaterials. 2020 Nov;258:120275
pubmed: 32798741
Hum Gene Ther. 2020 Feb;31(3-4):151-162
pubmed: 31914802
Sci Adv. 2020 Aug 21;6(34):
pubmed: 32937374
Nature. 2016 Jan 28;529(7587):490-5
pubmed: 26735016
Nat Methods. 2015 Apr;12(4):326-8
pubmed: 25730490
Neurochem Int. 2010 Nov;57(4):383-8
pubmed: 20350577
Mol Ther Methods Clin Dev. 2019 Oct 23;15:320-332
pubmed: 31788496
Front Mol Neurosci. 2020 Oct 22;13:583811
pubmed: 33192299
Neurosci Lett. 2019 Jan 10;689:11-13
pubmed: 29378296
Nat Commun. 2018 Feb 9;9(1):597
pubmed: 29426832
Neuropsychopharmacology. 2018 Jan;43(2):272-284
pubmed: 28462942
J Clin Invest. 2005 Jun;115(6):1449-57
pubmed: 15931380
J Control Release. 2016 Feb 28;224:136-145
pubmed: 26780172
Ann Clin Transl Neurol. 2020 Oct;7(10):1985-1995
pubmed: 32941707
Nat Commun. 2014 Feb 25;5:3338
pubmed: 24569435
Science. 2011 Sep 9;333(6048):1456-8
pubmed: 21778362
Mol Ther. 2020 Jun 3;28(6):1389-1391
pubmed: 32428441
Cell Res. 2018 Aug;28(8):855-861
pubmed: 29875396
Cell. 2015 Aug 27;162(5):1113-26
pubmed: 26317473
JAMA Neurol. 2018 Jul 1;75(7):834-841
pubmed: 29582053
Neurosci Lett. 2014 Apr 17;565:30-8
pubmed: 24406153
Mol Ther. 2011 Mar;19(3):526-35
pubmed: 21179009
Nature. 2015 Jan 29;517(7536):583-8
pubmed: 25494202
ACS Nano. 2020 Jan 28;14(1):360-371
pubmed: 31887011
Nucleic Acids Res. 2019 May 7;47(8):e44
pubmed: 30759231
Cell Stem Cell. 2019 Apr 4;24(4):551-565.e8
pubmed: 30905619
Biochem Biophys Res Commun. 2020 Dec 10;533(3):449-457
pubmed: 32972746
Gene Ther. 2005 Oct;12(20):1503-8
pubmed: 15944733
JCI Insight. 2017 Apr 6;2(7):e90133
pubmed: 28405611
J Neurosci. 2020 May 13;40(20):3896-3914
pubmed: 32300046
Nat Biotechnol. 2015 May;33(5):510-7
pubmed: 25849900
Lancet. 2014 Mar 29;383(9923):1138-46
pubmed: 24412048
Nat Commun. 2013;4:1770
pubmed: 23612311
Eur J Neurosci. 2010 Oct;32(8):1326-36
pubmed: 20846325
Mol Ther. 2020 May 6;28(5):1373-1380
pubmed: 32213322
Sci Rep. 2017 Aug 21;7(1):8943
pubmed: 28827551
ACS Chem Biol. 2018 Feb 16;13(2):383-388
pubmed: 28957631
Hum Gene Ther. 2012 Aug;23(8):903-14
pubmed: 22642214
Front Cell Neurosci. 2015 Feb 27;9:58
pubmed: 25774124
Cells. 2020 Dec 07;9(12):
pubmed: 33297340
Immunity. 2019 Jan 15;50(1):253-271.e6
pubmed: 30471926
Mol Ther. 2020 Feb 5;28(2):431-440
pubmed: 31843453
Sci Adv. 2019 Dec 06;5(12):eaay3255
pubmed: 31840076
Hum Mol Genet. 2020 Oct 10;29(17):2920-2935
pubmed: 32803224
Mol Ther. 2018 Nov 7;26(11):2638-2649
pubmed: 30266652
Nature. 2020 Sep;585(7825):397-403
pubmed: 32610343
Mol Ther. 2020 Apr 8;28(4):1177-1189
pubmed: 31991108
Front Cell Neurosci. 2020 Sep 10;14:570917
pubmed: 33132845
Proc Natl Acad Sci U S A. 2018 May 8;115(19):4903-4908
pubmed: 29686087
Cell. 2016 Sep 22;167(1):233-247.e17
pubmed: 27662091
Nat Med. 2018 Aug;24(8):1216-1224
pubmed: 30082871
Neuroreport. 2018 Dec 5;29(17):1504-1508
pubmed: 30334860
Nat Commun. 2020 Feb 27;11(1):1105
pubmed: 32107381
Nat Commun. 2019 Jan 3;10(1):53
pubmed: 30604771
Ann Neurol. 2015 Aug;78(2):248-57
pubmed: 26061140
Glia. 2020 Dec;68(12):2601-2612
pubmed: 32584476
Proc Natl Acad Sci U S A. 2000 Mar 28;97(7):3428-32
pubmed: 10688913
J Gene Med. 2014 Nov-Dec;16(11-12):364-73
pubmed: 25394283
J Control Release. 2021 Feb 10;330:587-598
pubmed: 33412229
Mol Ther Nucleic Acids. 2017 Sep 15;8:13-25
pubmed: 28918015
Cell Stem Cell. 2014 Feb 6;14(2):188-202
pubmed: 24360883
J Neurosurg. 2020 Jul 10;134(6):1751-1763
pubmed: 32915526
Nat Commun. 2019 Sep 30;10(1):4439
pubmed: 31570731
Nat Rev Mol Cell Biol. 2009 Mar;10(3):192-206
pubmed: 19234478
Handb Clin Neurol. 2018;148:693-700
pubmed: 29478608
Hum Gene Ther Clin Dev. 2018 Sep;29(3):148-155
pubmed: 30156440
Immunity. 2017 Sep 19;47(3):566-581.e9
pubmed: 28930663
Trends Neurosci. 2019 Dec;42(12):861-870
pubmed: 31706628
EMBO Mol Med. 2016 May 02;8(5):489-510
pubmed: 27025653
J Clin Hypertens. 1987 Dec;3(4):579-88
pubmed: 3330988
Curr Neuropharmacol. 2018;16(5):519-532
pubmed: 28847296
Colloids Surf B Biointerfaces. 2018 Oct 1;170:454-462
pubmed: 29958160
Nat Biotechnol. 2018 Sep;36(8):765-771
pubmed: 30010673
Nat Commun. 2016 May 10;7:11298
pubmed: 27161034
Mol Ther. 2009 Dec;17(12):2088-95
pubmed: 19672246
Nat Methods. 2015 Dec;12(12):1143-9
pubmed: 26501517
Science. 2020 Apr 17;368(6488):290-296
pubmed: 32217751
Sci Adv. 2020 Jul 15;6(29):eaba1773
pubmed: 32832622
Nat Biotechnol. 2018 Oct;36(9):843-846
pubmed: 29813047
Adv Exp Med Biol. 2017;958:91-127
pubmed: 28093710
Hum Gene Ther Clin Dev. 2014 Sep;25(3):164-77
pubmed: 25144894
Mol Ther Nucleic Acids. 2019 Jun 7;16:637-649
pubmed: 31108320
Nucleic Acids Res. 2014 Oct 29;42(19):e147
pubmed: 25122746
Glia. 2016 Dec;64(12):2181-2200
pubmed: 27685637
Nat Neurosci. 2017 Aug;20(8):1172-1179
pubmed: 28671695
Brain. 2020 Mar 1;143(3):960-975
pubmed: 32203581
Nat Biotechnol. 2017 May;35(5):444-452
pubmed: 28398344
Nat Neurosci. 2018 Apr;21(4):541-551
pubmed: 29507414
CNS Drugs. 2017 Feb;31(2):109-133
pubmed: 28101766
Nat Neurosci. 2019 Apr;22(4):524-528
pubmed: 30858603
Brain Res. 2020 May 1;1734:146738
pubmed: 32081534
Exp Neurol. 2018 Jun;304:125-131
pubmed: 29530713
Nat Biomed Eng. 2018 Jul;2(7):497-507
pubmed: 30948824
Nat Methods. 2015 May;12(5):401-403
pubmed: 25775043
BMC Genomics. 2020 Mar 17;21(1):239
pubmed: 32183699
Nat Neurosci. 2014 Dec;17(12):1720-7
pubmed: 25347353
J Vis Exp. 2019 Mar 29;(145):
pubmed: 30985756
Virology. 2010 Feb 5;397(1):167-75
pubmed: 19926109
Development. 2020 May 1;147(9):
pubmed: 32358096
Adv Sci (Weinh). 2020 Feb 06;7(6):1902312
pubmed: 32195078
J Neurosci. 2018 Mar 21;38(12):2923-2943
pubmed: 29431645
Gene Ther. 2016 Nov;23(11):807-814
pubmed: 27628693
Nat Biotechnol. 2016 Feb;34(2):204-9
pubmed: 26829320
Mol Ther Methods Clin Dev. 2016 Apr 13;3:16026
pubmed: 27308302
Adv Exp Med Biol. 2019;1175:355-381
pubmed: 31583595
Cells. 2020 Sep 27;9(10):
pubmed: 32992620
Hum Gene Ther. 2016 Dec;27(12):982-996
pubmed: 27604541
J Virol. 2001 Dec;75(24):12382-92
pubmed: 11711628
Hum Gene Ther. 2020 Dec;31(23-24):1237-1259
pubmed: 33233962
Cell Rep. 2017 Sep 19;20(12):2980-2991
pubmed: 28930690
Nature. 2019 May;569(7756):433-437
pubmed: 30995674
Mol Ther Methods Clin Dev. 2017 Apr 19;5:153-164
pubmed: 28497073
J Neurol. 2017 Oct;264(10):2068-2074
pubmed: 28821953
Curr Pharm Des. 2017;23(33):5010-5021
pubmed: 28641533
PLoS Pathog. 2018 May 3;14(5):e1006929
pubmed: 29723270
BMC Neurosci. 2010 Oct 18;11:132
pubmed: 20955577
Ann Neurol. 2018 Jan;83(1):27-39
pubmed: 29226998
Nature. 2018 Apr 5;556(7699):57-63
pubmed: 29512652
Nature. 2019 Dec;576(7785):149-157
pubmed: 31634902
Front Genet. 2016 Jan 12;6:362
pubmed: 26793235
J Gene Med. 2013 Jun-Jul;15(6-7):219-32
pubmed: 23658149
Proc Natl Acad Sci U S A. 2016 Jun 14;113(24):E3441-50
pubmed: 27247388
Int J Mol Sci. 2020 Oct 25;21(21):
pubmed: 33113845
Neurobiol Dis. 2020 Jul;141:104954
pubmed: 32445790
Front Immunol. 2020 Sep 25;11:559342
pubmed: 33101276
Hum Gene Ther Clin Dev. 2015 Jun;26(2):113-24
pubmed: 25758611
Glia. 2016 May;64(5):826-39
pubmed: 26847266
Sci Adv. 2019 May 08;5(5):eaax5717
pubmed: 31086823
Brain Res. 2020 Jul 15;1739:146832
pubmed: 32289279
Gene Ther. 2003 Apr;10(8):657-67
pubmed: 12692594
Neuroscience. 2019 Jan 1;396:73-78
pubmed: 30458223
Cell. 2014 Oct 23;159(3):635-46
pubmed: 25307933
Front Mol Neurosci. 2019 May 27;12:110
pubmed: 31191241
Gene Ther. 2021 Feb;28(1-2):75-88
pubmed: 32632267
PLoS One. 2013 Jun 25;8(6):e67680
pubmed: 23825679
Hum Mol Genet. 2015 Jun 15;24(12):3372-89
pubmed: 25749991
Glia. 2018 Jun;66(6):1244-1262
pubmed: 29076603
Nat Commun. 2018 Jul 17;9(1):2777
pubmed: 30018371
Nature. 2017 Nov 23;551(7681):464-471
pubmed: 29160308
Cell. 2013 Feb 28;152(5):1173-83
pubmed: 23452860
Mol Ther. 2018 Oct 3;26(10):2431-2442
pubmed: 30005866
Mol Ther Methods Clin Dev. 2018 Dec 06;12:111-122
pubmed: 30619914
J Neurosci. 2002 Jun 15;22(12):4885-96
pubmed: 12077186
Nat Biotechnol. 2017 May;35(5):431-434
pubmed: 28191903
J Neurosci Res. 2005 Nov 1;82(3):397-403
pubmed: 16158420
Nucleic Acids Res. 2017 Feb 28;45(4):1703-1713
pubmed: 27899645
Nat Commun. 2017 Jun 06;8:15790
pubmed: 28585549
Pediatr Neurol. 2020 Sep;110:5-19
pubmed: 32684374
Front Cell Neurosci. 2020 Sep 18;14:563446
pubmed: 33192310
Med Wieku Rozwoj. 2013 Oct-Dec;17(4):293-300
pubmed: 24519770
Alzheimers Dement. 2014 Sep;10(5):571-81
pubmed: 24411134
Ann Neurol. 2019 Nov;86(5):780-792
pubmed: 31433864
Alzheimers Dement. 2020 Dec;16(12):1613-1623
pubmed: 32881347
Annu Rev Immunol. 2017 Apr 26;35:441-468
pubmed: 28226226
Cell. 2013 Jul 18;154(2):442-51
pubmed: 23849981
Oncogene. 2018 Sep;37(38):5160-5174
pubmed: 29849122
Nat Biotechnol. 2018 Nov;36(10):977-982
pubmed: 30059493
J Neurochem. 2016 Jan;136 Suppl 1:49-62
pubmed: 25708596
Proc Natl Acad Sci U S A. 2008 Nov 18;105(46):17913-8
pubmed: 18997009
Nature. 2016 Apr 20;533(7603):420-4
pubmed: 27096365
Nat Commun. 2019 Sep 19;10(1):4284
pubmed: 31537810
Nat Rev Genet. 2018 Dec;19(12):770-788
pubmed: 30323312
Trends Immunol. 2020 Sep;41(9):794-804
pubmed: 32800704
Nat Commun. 2018 Sep 7;9(1):3651
pubmed: 30194297
Cell Res. 2017 Jun;27(6):801-814
pubmed: 28524166
Neurosci Lett. 2019 Aug 10;707:134310
pubmed: 31158432
Nat Commun. 2020 Jun 8;11(1):2876
pubmed: 32513994
Nat Biomed Eng. 2020 Jan;4(1):97-110
pubmed: 31937940
J Pharm Sci. 2020 Jan;109(1):62-67
pubmed: 31589876
Gene Ther. 2020 Jun;27(6):247-253
pubmed: 32203197
J Control Release. 2016 Jan 28;222:32-46
pubmed: 26658072
Nat Med. 2018 Oct;24(10):1519-1525
pubmed: 30297904
Nat Commun. 2020 Nov 30;11(1):6129
pubmed: 33257666
Glia. 2008 Apr;56(5):481-93
pubmed: 18240313
ACS Nano. 2020 Aug 25;14(8):9243-9262
pubmed: 32697075
Cells. 2019 Nov 12;8(11):
pubmed: 31726662
Hum Mol Genet. 2014 Jun 15;23(12):3250-68
pubmed: 24463623
Mol Ther. 2008 Oct;16(10):1710-8
pubmed: 18714307
Science. 2019 Apr 19;364(6437):292-295
pubmed: 30819931
PLoS One. 2018 Sep 13;13(9):e0203785
pubmed: 30212518
Cell Stem Cell. 2010 Oct 8;7(4):483-95
pubmed: 20887954
Neural Regen Res. 2021 Feb;16(2):270-280
pubmed: 32859774
Cell Rep. 2013 Jul 25;4(2):385-401
pubmed: 23850290
Nat Biotechnol. 2017 Apr;35(4):371-376
pubmed: 28191901
Nat Biotechnol. 2019 Oct;37(10):1145-1148
pubmed: 31548727
Nature. 2019 Jul;571(7764):275-278
pubmed: 31181567
Nat Med. 2019 Jul;25(7):1123-1130
pubmed: 31270503
Front Cell Neurosci. 2013 Mar 18;7:26
pubmed: 23507975
Cell Rep. 2020 Feb 4;30(5):1271-1281
pubmed: 32023447
Cell Stem Cell. 2018 Aug 02;23(2):239-251.e6
pubmed: 30075130
Glia. 2019 Dec;67(12):2221-2247
pubmed: 31429127
Int J Mol Sci. 2016 Sep 08;17(9):
pubmed: 27618019
Hum Gene Ther. 2008 May;19(5):463-74
pubmed: 18473686
Hum Mol Genet. 2010 Jun 1;19(11):2208-27
pubmed: 20203170
Mol Ther. 2012 Jul;20(7):1338-48
pubmed: 22434134
Sci Rep. 2017 Jul 20;7(1):6030
pubmed: 28729628
Front Immunol. 2020 Apr 17;11:670
pubmed: 32362898
Nucleic Acids Res. 2019 Sep 26;47(17):e99
pubmed: 31299082
PLoS Genet. 2010 Mar 05;6(3):e1000869
pubmed: 20221260
Gene Ther. 2003 Aug;10(17):1528-34
pubmed: 12900769
Nat Commun. 2017 Feb 21;8:14500
pubmed: 28220790
Nat Rev Neurol. 2019 Sep;15(9):540-555
pubmed: 31324897
Curr Pharm Des. 2016;22(22):3305-23
pubmed: 26845323
Glia. 2018 Feb;66(2):413-427
pubmed: 29119608
Nat Biotechnol. 2013 Sep;31(9):833-8
pubmed: 23907171
Cells. 2020 Sep 26;9(10):
pubmed: 32993090
Gene Ther. 2020 Apr;27(3-4):157-169
pubmed: 31819203
Front Mol Neurosci. 2018 Aug 15;11:282
pubmed: 30158855
J Neurosci. 2010 Mar 24;30(12):4508-14
pubmed: 20335488
Mult Scler Relat Disord. 2019 Feb;28:263-272
pubmed: 30639828
Neurochem Res. 2020 Nov;45(11):2732-2748
pubmed: 32910302
Nat Biotechnol. 2018 Jul;36(6):536-539
pubmed: 29702637
Front Cell Dev Biol. 2020 Nov 11;8:548283
pubmed: 33262983
Cell. 2016 Sep 22;167(1):219-232.e14
pubmed: 27662090
PLoS One. 2017 Dec 15;12(12):e0188830
pubmed: 29244806
Cell. 2018 Feb 22;172(5):979-992.e6
pubmed: 29456084
Biomaterials. 2019 Jul;209:25-40
pubmed: 31026609
Int J Mol Sci. 2020 Nov 30;21(23):
pubmed: 33266211
Nat Neurosci. 2018 Mar;21(3):440-446
pubmed: 29335603
Science. 2019 Apr 19;364(6437):289-292
pubmed: 30819928
Cell Rep. 2015 Oct 13;13(2):234-41
pubmed: 26440889