Treatment of Inherited Retinal Dystrophies with Somatic Cell Therapy Medicinal Product: A Review.
Stargardt disease
advanced therapy medicinal products
embryonic stem cells
good manufacturing practices
induced pluripotent stem cells
inherited retinal dystrophies
mesenchymal stromal cells
retinal organoids
retinal progenitor cells
retinitis pigmentosa
somatic cell therapy medicinal products
Journal
Life (Basel, Switzerland)
ISSN: 2075-1729
Titre abrégé: Life (Basel)
Pays: Switzerland
ID NLM: 101580444
Informations de publication
Date de publication:
09 May 2022
09 May 2022
Historique:
received:
22
04
2022
revised:
05
05
2022
accepted:
06
05
2022
entrez:
28
5
2022
pubmed:
29
5
2022
medline:
29
5
2022
Statut:
epublish
Résumé
Inherited retinal dystrophies and retinal degenerations related to more common diseases (i.e., age-related macular dystrophy) are a major issue and one of the main causes of low vision in pediatric and elderly age groups. Advancement and understanding in molecular biology and the possibilities raised by gene-editing techniques opened a new era for clinicians and patients due to feasible possibilities of treating disabling diseases and the reduction in their complications burden. The scope of this review is to focus on the state-of-the-art in somatic cell therapy medicinal products as the basis of new insights and possibilities to use this approach to treat rare eye diseases.
Identifiants
pubmed: 35629375
pii: life12050708
doi: 10.3390/life12050708
pmc: PMC9147057
pii:
doi:
Types de publication
Journal Article
Review
Langues
eng
Références
Anat Rec (Hoboken). 2014 Jan;297(1):137-60
pubmed: 24293400
Curr Opin Neurobiol. 2012 Oct;22(5):768-77
pubmed: 22405989
Biologics. 2021 Jul 27;15:299-306
pubmed: 34349498
N Engl J Med. 2004 Mar 25;350(13):1353-6
pubmed: 14999088
Lancet. 2015 Feb 7;385(9967):509-16
pubmed: 25458728
J Med Genet. 2003 Sep;40(9):641-50
pubmed: 12960208
Lancet. 2012 Feb 25;379(9817):713-20
pubmed: 22281388
Neuron. 2012 Oct 18;76(2):266-80
pubmed: 23083731
Mol Vis. 2011;17:1759-70
pubmed: 21750603
Retina. 2011 Jun;31(6):1207-14
pubmed: 21293313
Invest Ophthalmol Vis Sci. 2011 Nov 11;52(12):8785-90
pubmed: 21896853
World J Stem Cells. 2021 Jun 26;13(6):632-644
pubmed: 34249232
Stem Cells. 2017 May;35(5):1176-1188
pubmed: 28220575
PLoS One. 2011 Apr 29;6(4):e18992
pubmed: 21559507
Int J Mol Sci. 2017 Jul 28;18(8):
pubmed: 28788088
Dis Model Mech. 2015 May;8(5):421-7
pubmed: 26035859
Curr Opin Cell Biol. 2020 Feb;62:37-45
pubmed: 31518914
Zhonghua Yan Ke Za Zhi. 2004 Jul;40(7):448-52
pubmed: 15454058
Cytotherapy. 2005;7(5):393-5
pubmed: 16236628
Semin Ophthalmol. 2016;31(1-2):25-9
pubmed: 26959126
Clin Exp Reprod Med. 2011 Dec;38(4):216-21
pubmed: 22384445
Invest Ophthalmol Vis Sci. 2016 Apr 1;57(5):ORSFd1-9
pubmed: 27116661
Ann Anat. 2017 Mar;210:52-63
pubmed: 27986614
Nat Neurosci. 2001 Sep;4(9):877-86
pubmed: 11528418
J Clin Med. 2014 Dec 19;3(4):1511-41
pubmed: 26237613
Nature. 2012 May 3;485(7396):99-103
pubmed: 22522934
Sci Rep. 2017 Apr 10;7(1):766
pubmed: 28396597
Mol Vis. 2011 Feb 22;17:558-75
pubmed: 21364903
Cell Death Dis. 2020 Sep 23;11(9):793
pubmed: 32968042
Stem Cells Transl Med. 2017 Apr;6(4):1273-1285
pubmed: 28198592
Int J Mol Sci. 2019 Apr 10;20(7):
pubmed: 30974904
Prog Retin Eye Res. 2020 Mar;75:100779
pubmed: 31494256
Adv Exp Med Biol. 2018;1089:47-57
pubmed: 29774506
Cells. 2017 Feb 02;6(1):
pubmed: 28157165
Surv Ophthalmol. 2006 May-Jun;51(3):232-58
pubmed: 16644365
Pharmaceutics. 2021 Mar 06;13(3):
pubmed: 33800934
Stem Cell Res Ther. 2020 May 27;11(1):203
pubmed: 32460894
Cells. 2020 Apr 10;9(4):
pubmed: 32290105
Int J Clin Exp Med. 2014 Nov 15;7(11):3843-52
pubmed: 25550892
Prog Retin Eye Res. 2018 Sep;66:157-186
pubmed: 29597005
Stem Cells. 2006 Dec;24(12):2868-76
pubmed: 16902198
Graefes Arch Clin Exp Ophthalmol. 2008 Feb;246(2):255-65
pubmed: 18004585
Exp Eye Res. 2009 Sep;89(3):301-10
pubmed: 19524569
J Genet. 2009 Dec;88(4):417-24
pubmed: 20090205
Mol Vis. 2009;15:99-107
pubmed: 19156227
Nat Protoc. 2009;4(6):811-24
pubmed: 19444239
J Neurosci. 2003 Aug 27;23(21):7742-9
pubmed: 12944502
Cytotherapy. 2016 Apr;18(4):487-96
pubmed: 26897559
Exp Eye Res. 2005 Apr;80(4):515-25
pubmed: 15781279
J Neurosci Methods. 2010 Jun 30;190(1):63-70
pubmed: 20447416
Nat Protoc. 2007;2(12):3081-9
pubmed: 18079707
Stem Cells. 2006 Sep;24(9):2060-70
pubmed: 16644923
Int J Mol Sci. 2021 Sep 23;22(19):
pubmed: 34638582
Nat Biotechnol. 2000 Apr;18(4):399-404
pubmed: 10748519
Stem Cell Res Ther. 2016 Dec 1;7(1):178
pubmed: 27906070
Invest Ophthalmol Vis Sci. 2014 Dec 09;56(1):81-9
pubmed: 25491299
J Biomed Sci. 2018 Mar 30;25(1):31
pubmed: 29602309
Zentralbl Neurochir. 2005 May;66(2):75-8
pubmed: 15846535
Orphanet J Rare Dis. 2006 Oct 11;1:40
pubmed: 17032466
Ophthalmology. 2015 Feb;122(2):335-44
pubmed: 25444351
Proc Natl Acad Sci U S A. 2009 Sep 29;106(39):16698-703
pubmed: 19706890
Malays J Pathol. 2013 Jun;35(1):17-32
pubmed: 23817392
Stem Cells. 2011 May;29(5):825-35
pubmed: 21480547
Science. 2008 Aug 1;321(5889):699-702
pubmed: 18276851
Cytotherapy. 2006;8(4):315-7
pubmed: 16923606
Stem Cell Res Ther. 2021 Jan 19;12(1):69
pubmed: 33468246
Stem Cells. 2018 Sep;36(9):1329-1340
pubmed: 29770526
Exp Eye Res. 2018 Dec;177:65-75
pubmed: 30029023
Cell Transplant. 2014;23(9):1045-59
pubmed: 23676629
Tissue Eng Part A. 2009 Jun;15(6):1401-13
pubmed: 19113950
Cell Transplant. 2016;25(6):1145-57
pubmed: 26065854
Nature. 2006 Nov 9;444(7116):203-7
pubmed: 17093405
Nat Commun. 2015 Feb 19;6:6286
pubmed: 25695148
Stem Cells. 2009 Oct;27(10):2427-34
pubmed: 19658190
Cell Transplant. 2011;20(1):5-14
pubmed: 21396235
Nat Rev Nephrol. 2018 Aug;14(8):493-507
pubmed: 29895977
Invest Ophthalmol Vis Sci. 2010 Jul;51(7):3742-8
pubmed: 20207980
J Biol Chem. 2013 Nov 29;288(48):34484-93
pubmed: 24129572
Dev Dyn. 2003 Dec;228(4):664-71
pubmed: 14648843
Regen Med. 2019 May;14(4):321-329
pubmed: 30977436
Nat Commun. 2014 Jun 10;5:4047
pubmed: 24915161
Taiwan J Ophthalmol. 2021 Sep 11;11(3):205-206
pubmed: 34703734
Stem Cells. 2011 Aug;29(8):1206-18
pubmed: 21678528
Annu Rev Vis Sci. 2015 Nov 24;1:263-289
pubmed: 28532365
Proc Natl Acad Sci U S A. 2013 Jan 2;110(1):354-9
pubmed: 23248312
Int J Mol Sci. 2020 Oct 22;21(21):
pubmed: 33105778
Invest Ophthalmol Vis Sci. 2012 Sep 14;53(10):6282-90
pubmed: 22871841
Cell. 2007 Nov 30;131(5):861-72
pubmed: 18035408
Clin Exp Optom. 2021 May;104(4):444-454
pubmed: 33689657
Am J Hum Genet. 1999 Feb;64(2):422-34
pubmed: 9973280
Invest Ophthalmol Vis Sci. 2020 Jul 1;61(8):38
pubmed: 32721019
Exp Neurol. 2009 Apr;216(2):439-48
pubmed: 19320003
Int Ophthalmol Clin. 2021 Oct 1;61(4):157-165
pubmed: 34584053
Prog Retin Eye Res. 2019 Jul;71:1-25
pubmed: 30885665
Dev Biol. 2018 Jan 15;433(2):132-143
pubmed: 29291970
J Neurobiol. 2002 Feb 15;50(3):209-20
pubmed: 11810636
PLoS One. 2010 Jan 20;5(1):e8763
pubmed: 20098701
Exp Cell Res. 2015 Apr 10;333(1):11-20
pubmed: 25724900
Mol Med Rep. 2016 Oct;14(4):3104-12
pubmed: 27514011
Mol Med. 2012 Dec 06;18:1312-9
pubmed: 22895806
Angiogenesis. 2016 Apr;19(2):107-18
pubmed: 26880135
Cells. 2021 Mar 07;10(3):
pubmed: 33799995
Stem Cell Rev Rep. 2021 Aug;17(4):1154-1173
pubmed: 33410097
Nat Rev Mol Cell Biol. 2011 Feb;12(2):79-89
pubmed: 21252997
Biochem Biophys Res Commun. 2004 Apr 9;316(3):753-4
pubmed: 15033464
Invest Ophthalmol Vis Sci. 2004 Dec;45(12):4251-5
pubmed: 15557428
Proc Natl Acad Sci U S A. 2014 Jun 10;111(23):8518-23
pubmed: 24912154
Arq Bras Oftalmol. 2013 Mar-Apr;76(2):105-10
pubmed: 23828471
Invest Ophthalmol Vis Sci. 2016 Apr 1;57(5):ORSFc1-9
pubmed: 27116660
Mol Vis. 2010 Jul 14;16:1304-16
pubmed: 20664793