Mouse γ-Synuclein Promoter-Mediated Gene Expression and Editing in Mammalian Retinal Ganglion Cells.
Animals
CRISPR-Cas Systems
Dependovirus
/ genetics
Female
Gene Deletion
Gene Expression Regulation
/ genetics
Genetic Therapy
Humans
Induced Pluripotent Stem Cells
Male
Mice
Mice, Inbred C57BL
Optic Nerve
/ pathology
Optic Nerve Diseases
/ genetics
RNA Editing
/ genetics
Retinal Ganglion Cells
/ metabolism
Transgenes
/ genetics
gamma-Synuclein
/ genetics
AAV
CRSPR
RGC
Sncg
neuroprotection
optic nerve
Journal
The Journal of neuroscience : the official journal of the Society for Neuroscience
ISSN: 1529-2401
Titre abrégé: J Neurosci
Pays: United States
ID NLM: 8102140
Informations de publication
Date de publication:
13 05 2020
13 05 2020
Historique:
received:
14
01
2020
revised:
28
03
2020
accepted:
02
04
2020
pubmed:
18
4
2020
medline:
6
10
2020
entrez:
18
4
2020
Statut:
ppublish
Résumé
Optic neuropathies are a group of optic nerve (ON) diseases caused by various insults including glaucoma, inflammation, ischemia, trauma, and genetic deficits, which are characterized by retinal ganglion cell (RGC) death and ON degeneration. An increasing number of genes involved in RGC intrinsic signaling have been found to be promising neural repair targets that can potentially be modulated directly by gene therapy, if we can achieve RGC specific gene targeting. To address this challenge, we first used adeno-associated virus (AAV)-mediated gene transfer to perform a low-throughput
Identifiants
pubmed: 32300046
pii: JNEUROSCI.0102-20.2020
doi: 10.1523/JNEUROSCI.0102-20.2020
pmc: PMC7219295
doi:
Substances chimiques
gamma-Synuclein
0
Types de publication
Journal Article
Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't
Research Support, U.S. Gov't, Non-P.H.S.
Langues
eng
Sous-ensembles de citation
IM
Pagination
3896-3914Subventions
Organisme : NEI NIH HHS
ID : T32 EY027816
Pays : United States
Organisme : NEI NIH HHS
ID : P30 EY026877
Pays : United States
Organisme : NEI NIH HHS
ID : R01 EY023295
Pays : United States
Organisme : NEI NIH HHS
ID : R01 EY024984
Pays : United States
Organisme : NEI NIH HHS
ID : R01 EY026766
Pays : United States
Organisme : NEI NIH HHS
ID : U24 EY029903
Pays : United States
Organisme : NEI NIH HHS
ID : U01 EY027261
Pays : United States
Organisme : NEI NIH HHS
ID : K08 EY022058
Pays : United States
Organisme : NEI NIH HHS
ID : R01 EY024932
Pays : United States
Organisme : NEI NIH HHS
ID : R21 EY031063
Pays : United States
Organisme : NEI NIH HHS
ID : R01 EY025295
Pays : United States
Organisme : NEI NIH HHS
ID : R01 EY028106
Pays : United States
Informations de copyright
Copyright © 2020 the authors.
Références
Mol Cell Biol. 2015 Nov;35(22):3800-9
pubmed: 26370509
Hum Mol Genet. 2017 Oct 1;26(R2):R139-R150
pubmed: 28977448
J Integr Med. 2014 Nov;12(6):483-94
pubmed: 25412666
J Neurosci. 2008 Jan 9;28(2):548-61
pubmed: 18184797
J Neurobiol. 1994 Dec;25(12):1570-8
pubmed: 7861120
Nat Med. 2019 Feb;25(2):229-233
pubmed: 30664785
Hum Gene Ther. 2015 May;26(5):257-65
pubmed: 25807962
Science. 2012 Jul 27;337(6093):481-4
pubmed: 22678360
Lancet. 2009 Nov 7;374(9701):1597-605
pubmed: 19854499
Exp Neurol. 2013 Aug;246:54-61
pubmed: 22285251
Sci Rep. 2015 Nov 13;5:16595
pubmed: 26563826
Nature. 2015 Apr 9;520(7546):186-91
pubmed: 25830891
Nat Neurosci. 2019 Aug;22(8):1345-1356
pubmed: 31285614
Neural Plast. 2018 May 2;2018:7108948
pubmed: 29853847
J Comp Neurol. 2014 Apr 15;522(6):1411-43
pubmed: 24318667
Proc Natl Acad Sci U S A. 2010 Mar 30;107(13):5961-6
pubmed: 20231467
Hum Gene Ther. 2008 Oct;19(10):979-90
pubmed: 18774912
Mol Ther. 2011 Feb;19(2):293-301
pubmed: 21045809
EMBO J. 1990 Mar;9(3):833-40
pubmed: 1968831
J Comp Neurol. 2007 Jun 20;502(6):1047-65
pubmed: 17444492
Nat Biotechnol. 2013 Sep;31(9):827-32
pubmed: 23873081
Science. 2010 Jul 23;329(5990):413-7
pubmed: 20576849
Mol Ther. 2016 Mar;24(3):556-63
pubmed: 26666451
J Neurosci. 2016 May 25;36(21):5891-903
pubmed: 27225776
J Neurosci. 2013 Aug 14;33(33):13569-80
pubmed: 23946415
Lancet Neurol. 2014 Jan;13(1):83-99
pubmed: 24331795
J Integr Med. 2014 Jan;12(1):20-34
pubmed: 24461592
J Cell Biol. 2012 Jan 9;196(1):7-18
pubmed: 22232700
Exp Neurol. 2004 Mar;186(1):6-19
pubmed: 14980806
Hum Gene Ther. 2011 Jul;22(7):843-52
pubmed: 21126217
Eur J Pharm Biopharm. 2015 Sep;95(Pt B):343-52
pubmed: 25615882
J Neurosci Res. 2001 Jul 1;65(1):68-77
pubmed: 11433431
Nature. 2016 Jan 21;529(7586):326-35
pubmed: 26791723
Cell Death Dis. 2017 Jul 20;8(7):e2936
pubmed: 28726788
Mol Ther. 2007 Sep;15(9):1677-85
pubmed: 17565352
Brain. 2015 Jan;138(Pt 1):11-27
pubmed: 25433914
Nat Methods. 2016 Dec;13(12):1043-1049
pubmed: 27776111
Invest Ophthalmol Vis Sci. 2017 Dec 1;58(14):6091-6104
pubmed: 29204649
Curr Protoc Stem Cell Biol. 2015 Feb 02;32:1H.8.1-1H.8.20
pubmed: 25640818
Prog Retin Eye Res. 2016 Nov;55:149-181
pubmed: 27693724
Mol Vis. 2008 Aug 22;14:1540-8
pubmed: 18728752
Adv Exp Med Biol. 2017;983:147-157
pubmed: 28639197
Blood. 2002 Sep 1;100(5):1662-9
pubmed: 12176886
Vision Res. 2008 Feb;48(3):377-85
pubmed: 17950399
Elife. 2016 Mar 30;5:e14908
pubmed: 27026523
Front Biosci. 2003 Sep 01;8:s1140-56
pubmed: 12957857
Curr Opin Ophthalmol. 2018 May;29(3):234-238
pubmed: 29538182
Invest Ophthalmol Vis Sci. 2016 Jun 1;57(7):3470-6
pubmed: 27367513
J Virol. 2003 Mar;77(6):3495-504
pubmed: 12610125
Nat Protoc. 2017 Apr;12(4):828-863
pubmed: 28333914
Invest Ophthalmol Vis Sci. 2004 Aug;45(8):2503-13
pubmed: 15277470
Neuroscience. 1994 Nov;63(2):591-602
pubmed: 7891867
Mol Cell Biol. 1991 Apr;11(4):2216-28
pubmed: 1672442
Hum Mol Genet. 2017 Aug 1;26(R1):R21-R27
pubmed: 28505344
Neuron. 2015 Jun 17;86(6):1369-84
pubmed: 26087164
Cell. 2013 Feb 28;152(5):1173-83
pubmed: 23452860
N Engl J Med. 2008 May 22;358(21):2240-8
pubmed: 18441370
Neuron. 2012 Feb 9;73(3):445-52
pubmed: 22325198
Mol Ther. 2017 Nov 1;25(11):2546-2560
pubmed: 28807567
J Neurophysiol. 2016 Aug 1;116(2):602-10
pubmed: 27169509
Nat Rev Neurosci. 2014 Jun;15(6):394-409
pubmed: 24840802
Virology. 2003 Jun 20;311(1):89-95
pubmed: 12832206
Science. 2008 Nov 7;322(5903):963-6
pubmed: 18988856
Stem Cells. 2016 Jun;34(6):1553-62
pubmed: 26996528
Cell. 2004 Nov 12;119(4):567-78
pubmed: 15537545
Mol Ther. 2011 Mar;19(3):442-9
pubmed: 21081906
Ann Neurol. 2010 Jun;67(6):749-60
pubmed: 20517936
Nat Biotechnol. 2019 Mar;37(3):224-226
pubmed: 30809026
Invest Ophthalmol Vis Sci. 2010 Feb;51(2):1052-8
pubmed: 19737887
Neurophotonics. 2015 Jul;2(3):031209
pubmed: 26839901
Clin Transl Sci. 2017 Jul;10(4):242-248
pubmed: 28383804
Ophthalmology. 2017 Nov;124(11):1621-1634
pubmed: 28647203
J Neurosci. 2008 Mar 12;28(11):2735-44
pubmed: 18337403
Neuron. 2016 Feb 3;89(3):449-60
pubmed: 26844829
Sci Rep. 2017 Mar 31;7:45329
pubmed: 28361998
Ophthalmology. 2016 Mar;123(3):558-70
pubmed: 26606867
N Engl J Med. 2008 May 22;358(21):2231-9
pubmed: 18441371
J Neurosci. 2016 May 25;36(21):5785-98
pubmed: 27225768
Sci Rep. 2018 Sep 28;8(1):14520
pubmed: 30266927
Neuron. 1988 Nov;1(9):791-803
pubmed: 2908449
Mol Ther. 2013 Mar;21(3):509-19
pubmed: 23358189
Nat Rev Genet. 2011 May;12(5):341-55
pubmed: 21499295
Nat Commun. 2017 Mar 14;8:14716
pubmed: 28291770
Proc Natl Acad Sci U S A. 1996 Nov 12;93(23):13250-5
pubmed: 8917577
Nat Commun. 2014 Nov 10;5:5416
pubmed: 25382660
EBioMedicine. 2016 Aug;10:258-68
pubmed: 27426279
Cell. 2014 Oct 23;159(3):647-61
pubmed: 25307932
Science. 2013 Feb 15;339(6121):823-6
pubmed: 23287722
Nat Protoc. 2013 Nov;8(11):2281-2308
pubmed: 24157548
Dev Cell. 2014 Sep 8;30(5):513-27
pubmed: 25155555
Proc Natl Acad Sci U S A. 2009 Aug 18;106(33):13939-44
pubmed: 19666516
Science. 2013 Feb 15;339(6121):819-23
pubmed: 23287718
Nat Commun. 2016 Feb 04;7:10472
pubmed: 26843334
Stem Cells Transl Med. 2017 Nov;6(11):1972-1986
pubmed: 29024560
Elife. 2013 Jan 29;2:e00471
pubmed: 23386978
Genes Dev. 2015 Jul 15;29(14):1576-85
pubmed: 26178787
Nat Biotechnol. 2017 Nov 9;35(11):998-999
pubmed: 29121014