Emerging Gene Therapy Technologies for Retinal Ganglion Cell Neuroprotection.
Journal
Journal of neuro-ophthalmology : the official journal of the North American Neuro-Ophthalmology Society
ISSN: 1536-5166
Titre abrégé: J Neuroophthalmol
Pays: United States
ID NLM: 9431308
Informations de publication
Date de publication:
01 09 2023
01 09 2023
Historique:
pmc-release:
01
09
2024
medline:
22
8
2023
pubmed:
13
7
2023
entrez:
13
7
2023
Statut:
ppublish
Résumé
Optic neuropathies encompass a breadth of diseases that ultimately result in dysfunction and/or loss of retinal ganglion cells (RGCs). Although visual impairment from optic neuropathies is common, there is a lack of effective clinical treatments. Addressing a critical need for novel interventions, preclinical studies have been generating a growing body of evidence that identify promising new drug-based and cell-based therapies. Gene therapy is another emerging therapeutic field that offers the potential of specifically and robustly increasing long-term RGC survival in optic neuropathies. Gene therapy offers additional benefits of driving improvements following a single treatment administration, and it can be designed to target a variety of pathways that may be involved in individual optic neuropathies or across multiple etiologies. This review explores the history of gene therapy, the fundamentals of its application, and the emerging development of gene therapy technology as it relates to treatment of optic neuropathies.
Identifiants
pubmed: 37440418
doi: 10.1097/WNO.0000000000001955
pii: 00041327-990000000-00413
pmc: PMC10527513
mid: NIHMS1910364
doi:
Types de publication
Review
Journal Article
Research Support, Non-U.S. Gov't
Research Support, N.I.H., Extramural
Langues
eng
Sous-ensembles de citation
IM
Pagination
330-340Subventions
Organisme : NEI NIH HHS
ID : K08 EY030163
Pays : United States
Informations de copyright
Copyright © 2023 by North American Neuro-Ophthalmology Society.
Déclaration de conflit d'intérêts
A. G. Ross and K. S. Shindler hold intellectual property relevant to gene therapy and receive research funding from Gyroscope Therapeutics. D. K. Camacho, C. C. Go, B. Chaqour report no conflicts of interest.
Références
Surv Ophthalmol. 1999 Jun;43 Suppl 1:S142-50
pubmed: 10416757
Front Neurol. 2021 May 26;12:651639
pubmed: 34122299
Mol Ther Methods Clin Dev. 2022 Dec 14;28:129-145
pubmed: 36654798
J Neuroophthalmol. 2021 Sep 1;41(3):309-315
pubmed: 34415265
Hum Gene Ther Clin Dev. 2019 Sep;30(3):102-113
pubmed: 30968714
Proc Natl Acad Sci U S A. 2013 Feb 5;110(6):E517-25
pubmed: 23341635
Am J Ophthalmol. 2018 Aug;192:xv-xxii
pubmed: 29702074
J Clin Invest. 2008 Sep;118(9):3132-42
pubmed: 18688285
Lancet. 2016 Aug 13;388(10045):661-72
pubmed: 27375040
J Neurosci. 2002 May 15;22(10):3977-86
pubmed: 12019317
Mol Vis. 2007 Jan 05;13:1-11
pubmed: 17242675
N Engl J Med. 2011 Aug 25;365(8):725-33
pubmed: 21830940
Ophthalmology. 2008 Jun;115(6):1079-1082.e5
pubmed: 17976727
Mol Ther. 2012 Jun;20(6):1177-86
pubmed: 22453768
PLoS Genet. 2005 Jul;1(1):17-26
pubmed: 16103918
Nat Biotechnol. 2015 Mar;33(3):217-8
pubmed: 25748892
Neuron. 1996 Sep;17(3):401-11
pubmed: 8816704
Prog Retin Eye Res. 2006 Sep;25(5):490-513
pubmed: 16962364
Eur J Neurosci. 1998 Aug;10(8):2751-6
pubmed: 9767407
Cell. 2006 Dec 15;127(6):1109-22
pubmed: 17112576
Biomolecules. 2022 Jun 14;12(6):
pubmed: 35740955
Nature. 2020 Mar;579(7798):185
pubmed: 32157225
Drugs. 2016 May;76(7):805-13
pubmed: 27071925
J Neurosci. 2020 May 13;40(20):3896-3914
pubmed: 32300046
Nat Rev Drug Discov. 2019 May;18(5):358-378
pubmed: 30710128
J Biol Chem. 2005 Apr 22;280(16):16456-60
pubmed: 15716268
Front Neurol. 2021 May 24;12:662838
pubmed: 34108929
J Neurosci. 1994 Jul;14(7):4368-74
pubmed: 8027784
J Ophthalmol. 2019 Jan 15;2019:1803619
pubmed: 30766729
Surv Ophthalmol. 2010 Jan-Feb;55(1):47-63
pubmed: 20006051
Hum Gene Ther. 2018 Nov;29(11):1235-1241
pubmed: 30132368
Signal Transduct Target Ther. 2021 Feb 8;6(1):53
pubmed: 33558455
J Neuroophthalmol. 2013 Jun;33(2):172-8
pubmed: 23681241
Proc Natl Acad Sci U S A. 2012 Dec 4;109(49):20065-70
pubmed: 23129651
Ophthalmology. 2021 Jun;128(6):928-937
pubmed: 33161071
Invest Ophthalmol Vis Sci. 2000 Mar;41(3):764-74
pubmed: 10711692
Hum Gene Ther. 2020 Dec;31(23-24):1288-1299
pubmed: 32948113
Proc Natl Acad Sci U S A. 2019 Mar 19;116(12):5785-5794
pubmed: 30833387
AAPS J. 2006 Oct 06;8(4):E632-43
pubmed: 17233528
Trends Biotechnol. 2013 Jul;31(7):397-405
pubmed: 23664777
Sci Rep. 2020 Oct 5;10(1):16515
pubmed: 33020509
Hum Gene Ther. 2002 Jan 1;13(1):163-75
pubmed: 11779420
Invest Ophthalmol Vis Sci. 1994 Mar;35(3):907-15
pubmed: 8125754
PLoS One. 2015 Nov 11;10(11):e0142067
pubmed: 26560713
Discov Med. 2015 Jan;19(102):49-57
pubmed: 25636961
Lancet Neurol. 2014 Jan;13(1):83-99
pubmed: 24331795
Cell Death Dis. 2018 Sep 26;9(10):1007
pubmed: 30258047
Gene Ther. 2006 Sep;13(18):1328-41
pubmed: 16708079
Neurotoxicology. 2005 Oct;26(5):935-41
pubmed: 16126273
Front Neurol. 2022 Aug 12;13:964197
pubmed: 36034312
Adv Ther. 2016 Jun;33(6):959-81
pubmed: 27138604
Mol Ther. 2022 Apr 6;30(4):1421-1431
pubmed: 35114390
Dis Mon. 2014 Jun;60(6):276-81
pubmed: 24906673
J Clin Invest. 2012 May;122(5):1667-76
pubmed: 22523064
Invest Ophthalmol Vis Sci. 2007 Aug;48(8):3602-9
pubmed: 17652729
Sci Transl Med. 2020 Dec 9;12(573):
pubmed: 33298565
Curr Eye Res. 2021 May;46(5):710-718
pubmed: 33107352
Curr Neurol Neurosci Rep. 2022 Dec;22(12):881-892
pubmed: 36414808
Neurotherapeutics. 2019 Oct;16(4):1269-1282
pubmed: 31420820
Korean J Neurotrauma. 2018 Oct;14(2):55-60
pubmed: 30402419
J Immunol. 2016 Dec 15;197(12):4626-4638
pubmed: 27849168
J Neurosci. 1996 Jul 1;16(13):4186-94
pubmed: 8753880
Cell Death Dis. 2021 Aug 10;12(8):781
pubmed: 34376637
Nat Biotechnol. 2017 Jun 7;35(6):491-492
pubmed: 28591128
Sci Transl Med. 2014 Mar 12;6(227):227ra33
pubmed: 24622513
Nat Rev Genet. 2014 Aug;15(8):541-55
pubmed: 25022906
Invest Ophthalmol Vis Sci. 2018 Mar 1;59(3):1212-1220
pubmed: 29494741
Brain. 2010 Sep;133(9):2612-25
pubmed: 20495185
Nucleic Acid Ther. 2014 Aug;24(4):258-66
pubmed: 25054518
In Vivo. 2017 Jul-Aug;31(4):511-525
pubmed: 28652416
Drug Discov Today. 2019 Aug;24(8):1685-1693
pubmed: 31173914
Front Neurol. 2012 May 24;3:84
pubmed: 22654783
Gene Ther. 2021 May;28(5):256-264
pubmed: 33589779
Trends Endocrinol Metab. 2009 Sep;20(7):325-31
pubmed: 19713122
Invest Ophthalmol Vis Sci. 1998 Aug;39(9):1713-20
pubmed: 9699561
N Engl J Med. 2008 May 22;358(21):2240-8
pubmed: 18441370
Mol Cell Neurosci. 2012 Aug;51(1-2):53-9
pubmed: 22836101
Trans Am Ophthalmol Soc. 2000;98:223-32; discussion 232-5
pubmed: 11190025
Mol Ther. 2015 Mar;23(3):414-22
pubmed: 25515709
Proc Natl Acad Sci U S A. 1994 Mar 1;91(5):1632-6
pubmed: 8127857
Arch Ophthalmol. 2000 Apr;118(4):481-8
pubmed: 10766133
JAMA. 2014 May 14;311(18):1901-11
pubmed: 24825645
Neurosurg Rev. 2022 Jun;45(3):1895-1913
pubmed: 35034261
Invest Ophthalmol Vis Sci. 1995 Apr;36(5):774-86
pubmed: 7706025
Hum Gene Ther Clin Dev. 2013 Jun;24(2):55-64
pubmed: 23808604
Am J Ophthalmol. 2018 Sep;193:130-142
pubmed: 29940166
Hum Gene Ther. 2020 Jul;31(13-14):695-696
pubmed: 32605399
Cell Death Differ. 1999 Jul;6(7):673-82
pubmed: 10453078
Am J Ophthalmol. 2011 Jun;151(6):928-933.e2
pubmed: 21529765
Eur J Hum Genet. 2007 Oct;15(10):1079-89
pubmed: 17406640
J Gene Med. 2018 May;20(5):e3015
pubmed: 29575374
Mol Ther Methods Clin Dev. 2021 Oct 01;23:307-318
pubmed: 34729378
Int J Mol Sci. 2020 Aug 29;21(17):
pubmed: 32872441
Neurotherapeutics. 2023 Apr;20(3):896-907
pubmed: 36941497
Hum Gene Ther. 2017 Apr;28(4):314-322
pubmed: 28293963
Am J Hum Genet. 1996 Aug;59(2):481-5
pubmed: 8755941
Invest Ophthalmol Vis Sci. 1996 Mar;37(4):489-500
pubmed: 8595949
Gene Ther. 2009 Jul;16(7):862-73
pubmed: 19440225
Arch Ophthalmol. 2002 Jun;120(6):701-13; discussion 829-30
pubmed: 12049574
Dtsch Arztebl Int. 2015 Sep 11;112(37):616-25; quiz 626
pubmed: 26396053
Exp Eye Res. 2006 Aug;83(2):255-62
pubmed: 16546168
Clin Ophthalmol. 2007 Sep;1(3):233-46
pubmed: 19668477
Mol Neurobiol. 2020 Apr;57(4):1952-1965
pubmed: 31900864
Ophthalmology. 2021 May;128(5):649-660
pubmed: 33451738
J Neurobiol. 1994 Apr;25(4):431-8
pubmed: 8077968
JAMA Ophthalmol. 2019 Oct 1;137(10):1123-1124
pubmed: 31318388
JAMA Ophthalmol. 2019 Oct 01;137(10):1115-1123
pubmed: 31318398
N Engl J Med. 1992 Feb 27;326(9):581-8
pubmed: 1734247
Arch Ophthalmol. 1983 Jul;101(7):1059-68
pubmed: 6870629
Hum Gene Ther. 1996 Nov 10;7(17):2101-12
pubmed: 8934224
Hum Gene Ther. 2006 Aug;17(8):845-58
pubmed: 16942444
Front Neurosci. 2017 Sep 21;11:521
pubmed: 28983234
Front Cell Neurosci. 2012 Dec 31;6:63
pubmed: 23293585
Hum Gene Ther. 2005 May;16(5):541-50
pubmed: 15916479
Antioxidants (Basel). 2021 Dec 05;10(12):
pubmed: 34943051
Mol Ther. 2005 Mar;11(3):373-81
pubmed: 15727933
J Clin Invest. 2009 Apr;119(4):964-75
pubmed: 19307726
Arch Ophthalmol. 2002 Oct;120(10):1268-79
pubmed: 12365904
Science. 1995 Oct 20;270(5235):475-80
pubmed: 7570001
Sci Transl Med. 2011 Jun 22;3(88):88ra54
pubmed: 21697530
Mol Ther. 2002 May;5(5 Pt 1):509-16
pubmed: 11991741
FASEB J. 2006 Feb;20(2):389-91
pubmed: 16352651
J Med Ethics. 2001 Jun;27(3):148-50
pubmed: 11417019
Nature. 1968 Aug 17;219(5155):749-51
pubmed: 5667075
Indian J Ophthalmol. 2021 Feb;69(2):227-235
pubmed: 33463564
J Med Genet. 2002 Mar;39(3):162-9
pubmed: 11897814
J Biol Chem. 2001 Dec 7;276(49):46326-32
pubmed: 11574549
Invest Ophthalmol Vis Sci. 2000 Oct;41(11):3460-6
pubmed: 11006239