参考文献/References:
[1] VELERI S,LAZAR C H,CHANG B,SIEVING P A,BANIN E,SWAROOP A.Biology and therapy of inherited retinal degenerative disease: insights from mouse models[J].Dis Model Mech,2015,8(2):109-129.
[2] JONES M K,LU B,GIRMAN S,WANG S.Cell-based therapeutic strategies for replacement and preservation in retinal degenerative diseases [J].Prog Retin Eye Res,2017,58:1-27.
[3] FULLER-CARTER P I,BASIRI H,HARVEY A R,CARVALHO L S.Focused update on AAV-based gene therapy clinical trials for inherited retinal degeneration[J].BioDrugs,2020,34(6):763-781.
[4] ROSS M,OFRI R.The future of retinal gene therapy:evolving from subretinal to intravitreal vector delivery [J].Neural Regen Res,2021,16(9):1751-1759.
[5] UYAMA H,MANDAI M,TAKAHASHI M.Stem-cell-based therapies for retinal degenerative diseases:current challenges in the establishment of new treatment strategies [J].Dev Growth Differ,2021,63(1):59-71.
[6] ZARBIN M.Cell-based therapy for degenerative retinal disease[J].Trends Mol Med,2016,22(2):115-134.
[7] MITJAVILA-GARCIA M T,SIMONIN C,PESCHANSKI M.Embryonic stem cells:meeting the needs for cell therapy [J].Adv Drug Deliv Rev,2005,57(13):1935-1943.
[8] CLEVERS H.Stem cells.What is an adult stem cell? [J].Science,2015,350(6266):1319-1320.
[9] GURUSAMY N,ALSAYARI A,RAJASINGH S,RAJASINGH J.Adult stem cells for regenerative therapy [J].Prog Mol Biol Transl Sci,2018,160:1-22.
[10] MILES A,TROPEPE V.Retinal stem cell ‘retirement plans’:growth,regulation and species adaptations in the retinal ciliary marginal zone [J].Int J Mol Sci,2021,22(12):22(12):6528.
[11] SATARIAN L,NOURINIA R,SAFI S,KANAVI M R,JARUGHI N,DAFTARIAN N,et al.Intravitreal injection of bone marrow mesenchymal stem cells in patients with advanced retinitis pigmentosa; a safety study [J].J Ophthalmic Vis Res,2017,12(1):58-64.
[12] TUEKPRAKHON A,SANGKITPORN S,TRINAVARAT A,PAWESTRI A R,VAMVANIJ V,RUANGCHAINIKOM M,et al.Intravitreal autologous mesenchymal stem cell transplantation:a non-randomized phase I clinical trial in patients with retinitis pigmentosa [J].Stem Cell Res Ther,2021,12(1):52.
[13] ONER A,GONEN Z B,SINIM N,CETIN M,OZKUL Y.Subretinal adipose tissue-derived mesenchymal stem cell implantation in advanced stage retinitis pigmentosa:a phase I clinical safety study [J].Stem Cell Res Ther,2016,7(1):178.
[14] LUND R D,WANG S,LU B,GIRMAN S,HOLMES T,SAUVE Y,et al.Cells isolated from umbilical cord tissue rescue photoreceptors and visual functions in a rodent model of retinal disease [J].Stem Cells,2007,25(3):602-611.
[15] ZHAO T,LIANG Q,MENG X,DUAN P,WANG F,LI S,et al.Intravenous infusion of umbilical cord mesenchymal stem cells maintains and partially improves visual function in patients with advanced retinitis pigmentosa [J].Stem Cells Dev,2020,29(16):1029-1037.
[16] SPTER T,ASSUNO M,LIT K K,GONG G,WANG X,CHEN Y Y,et al.Engineering microparticles based on solidified stem cell secretome with an augmented pro-angiogenic factor portfolio for therapeutic angiogenesis [J].Bioact Mater,2022,17:526-541.
[17] SANCHEZ ALVARADO A,TSONIS P A.Bridging the regeneration gap:genetic insights from diverse animal models [J].Nat Rev Genet,2006,7(11):873-884.
[18] TROPEPE V,COLES B L,CHIASSON B J,HORSFORD D J,ELIA A J,MCINNES R R,et al.Retinal stem cells in the adult mammalian eye [J].Science,2000,287(5460):2032-2036.
[19] ALADDAD A M,KADOR K E.Adult stem cells,tools for repairing the retina [J].Curr Ophthalmol Rep,2019,7(1):21-29.
[20] JORSTAD N L,WILKEN M S,GRIMES W N,WOHL S G,VANDENBOSCH L S,YOSHIMATSU T,et al.Stimulation of functional neuronal regeneration from Muller glia in adult mice [J].Nature,2017,548(7665):103-107.
[21] WEBSTER M K,COOLEY-THEMM C A,BARNETT J D,BACH H B,VAINNER J M,WEBSTER S E,et al.Evidence of BrdU-positive retinal neurons after application of an Alpha7 nicotinic acetylcholine receptor agonist [J].Neuroscience,2017,346:437-446.
[22] ABDUL-AL M,KYEREMEH G K,SAEINASAB M,HEIDARI KESHEL S,SEFAT F.Stem cell niche microenvironment:review [J].Bioengineering (Basel),2021,8(8):108.
[23] LIU Y,CHEN S J,LI S Y,QU L H,MENG X H,WANG Y,et al.Long-term safety of human retinal progenitor cell transplantation in retinitis pigmentosa patients [J].Stem Cell Res Ther,2017,8(1):209.
[24] CUENCA N,FERNANDEZ-SANCHEZ L,MCGILL T J,LU B,WANG S,LUND R,et al.Phagocytosis of photoreceptor outer segments by transplanted human neural stem cells as a neuroprotective mechanism in retinal degeneration [J].Invest Ophthalmol Vis Sci,2013,54(10):6745-6756.
[25] NISHIDA A,TAKAHASHI M,TANIHARA H,NAKANO I,TAKAHASHI J B,MIZOGUCHI A,et al.Incorporation and differentiation of hippocampus-derived neural stem cells transplanted in injured adult rat retina [J].Invest Ophthalmol Vis Sci,2000,41(13):4268-4274.
[26] YOSHIHARA M,OGUCHI A,MURAKAWA Y.Genomic instability of iPSCs and challenges in their clinical applications [J].Adv Exp Med Biol,2019,1201:23-47.
[27] BRACHA P,MOORE N A,CIULLA T A.Induced pluripotent stem cell-based therapy for age-related macular degeneration [J].Expert Opin Biol Ther,2017,17(9):1113-1126.
[28] WANG Y,TANG Z,GU P.Stem/progenitor cell-based transplantation for retinal degeneration:a review of clinical trials [J].Cell Death Dis,2020,11(9):793.
[29] MAJJI A B,DE JUAN E,Jr.Retinal pigment epithelial autotransplantation:morphological changes in retina and choroid [J].Graefes Arch Clin Exp Ophthalmol,2000,238(9):779-791.
[30] ZHANG T,HU Y,LI Y,WU J,ZHAO L,WANG C,et al.Photoreceptors repair by autologous transplantation of retinal pigment epithelium and partial-thickness choroid graft in rabbits [J].Invest Ophthalmol Vis Sci,2009,50(6):2982-2988.
[31] DING X,ZHANG T,HONG Y,CHANG X,LI Y,MA Z,et al.Long-term ultrastructural outcomes of autologous transplantation of retinal pigment epithelium-partial thickness choroid(RPE-PTC) sheet in rabbits [J].Front Biosci (Landmark Ed),2022,27(3):106.
[32] BORAL S K,AGARWAL D,DAS A,SINHA T K.Long-term clinical outcomes of submacular blood removal with isolated autologous retinal pigment epithelium-choroid patch graft transplantation in long-standing large-sized submacular hematomas:an Indian experience [J].Indian J Ophthalmol,2020,68(10):2148-2153.
[33] PAROLINI B,DI SALVATORE A,PINACKATT S J,BALDI A,BESOZZI G,FINZI A,et al.Long-term results of autologous retinal pigment epithelium and choroid transplantation for the treatment of exudative and atrophic maculopathies [J].Retina,2020,40(3):507-520.
[34] TEZEL T H,DEL PRIORE L V,BERGER A S,KAPLAN H J.Adult retinal pigment epithelial transplantation in exudative age-related macular degeneration [J].Am J Ophthalmol,2007,143(4):584-595.
[35] ALGVERE P V,BERGLIN L,GOURAS P,SHENG Y.Transplantation of fetal retinal pigment epithelium in age-related macular degeneration with subfoveal neovascularization [J].Graefes Arch Clin Exp Ophthalmol,1994,232(12):707-716.
[36] LIDGERWOOD G E,SENABOUTH A,SMITH-ANTTILA C J A,GNANASAMBANDAPILLAI V,KACZOROWSKI D C,AMANN-ZALCENSTEIN D,et al.Transcriptomic profiling of human pluripotent stem cell-derived retinal pigment epithelium over time [J].Genomics Proteomics Bioinformatics,2021,19(2):223-242.
[37] AL-ANI A,SUNBA S,HAFEEZ B,TOMS D,UNGRIN M.In vitro maturation of retinal pigment epithelium is essential for maintaining high expression of key functional genes [J].Int J Mol Sci,2020,21(17):6066.
[38] VIHERIALA T,HONGISTO H,SORVARI J,SKOTTMAN H,NYMARK S,ILMARINEN T.Cell maturation influences the ability of hESC-RPE to tolerate cellular stress [J].Stem Cell Res Ther,2022,13(1):30.
[39] SCHWARTZ S D,HUBSCHMAN J P,HEILWELL G,FRANCO-CARDENAS V,PAN C K,OSTRICK R M,et al.Embryonic stem cell trials for macular degeneration:a preliminary report [J].Lancet,2012,379(9817):713-720.
[40] SUNG Y,LEE M J,CHOI J,JUNG S Y,CHONG S Y,SUNG J H,et al.Long-term safety and tolerability of subretinal transplantation of embryonic stem cell-derived retinal pigment epithelium in Asian Stargardt disease patients [J].Br J Ophthalmol,2021,105(6):829-837.
[41] DA CRUZ L,FYNES K,GEORGIADIS O,KERBY J,LUO Y H,AHMADO A,et al.Phase 1 clinical study of an embryonic stem cell-derived retinal pigment epithelium patch in age-related macular degeneration [J].Nat Biotechnol,2018,36(4):328-337.
[42] HSIUNG J,ZHU D,HINTON D R.Polarized human embryonic stem cell-derived retinal pigment epithelial cell monolayers have higher resistance to oxidative stress-induced cell death than nonpolarized cultures [J].Stem Cells Transl Med,2015,4(1):10-20.
[43] THOMAS B B,LIN B,MARTINEZ-CAMARILLO J C,ZHU D,MCLELLAND B T,NISTOR G,et al.Co-grafts of human embryonic stem cell derived retina organoids and retinal pigment epithelium for retinal reconstruction in immunodeficient retinal degenerate royal college of surgeons rats [J].Front Neurosci,2021,15:752958.
[44] RU L,WU N,WEI K,ZENG Y,LI Q,WENG C,et al.Improving cell survival and engraftment in vivo via layer-by-layer nanocoating of hESC-derived RPE cells [J].Stem Cell Res Ther,2020,11(1):495.
[45] SHRESTHA R,WEN Y T,TSAI R K.Effective differentiation and biological characterization of retinal pigment epithelium derived from human induced pluripotent stem cells [J].Curr Eye Res,2020,45(9):1155-1167.
[46] VOISIN A,MONVILLE C,PLANCHERON A,BALBOUS A,GAILLARD A,LEVEZIEL N.hRPE cells derived from induced pluripotent stem cells are more sensitive to oxidative stress than ARPE-19cells [J].Exp Eye Res,2018,177:76-86.
[47] MANDAI M,WATANABE A,KURIMOTO Y,HIRAMI Y,MORINAGA C,DAIMON T,et al.Autologous induced stem-cell-derived retinal cells for macular degeneration [J].N Engl J Med,2017,376(11):1038-1046.
[48] TAKAGI S,MANDAI M,GOCHO K,HIRAMI Y,YAMAMOTO M,FUJIHARA M,et al.Evaluation of transplanted autologous induced pluripotent stem cell-derived retinal pigment epithelium in exudative age-related macular degeneration [J].Ophthalmol Retina,2019,3(10):850-859.
[49] SHARMA R,KHRISTOV V,RISING A,JHA B S,DEJENE R,HOTALING N,et al.Clinical-grade stem cell-derived retinal pigment epithelium patch rescues retinal degeneration in rodents and pigs [J].Sci Transl Med,2019,11(475):eaat5580.
[50] YANG J M,CHUNG S,YUN K,KIM B,SO S,KANG S,et al.Long-term effects of human induced pluripotent stem cell-derived retinal cell transplantation in Pde6b knockout rats [J].Exp Mol Med,2021,53(4):631-642.
[51] CALEJO M T,SAARI J,VUORENPAA H,VUORIMAA-LAUKKANEN E,KALLIO P,AALTO-SETALA K,et al.Co-culture of human induced pluripotent stem cell-derived retinal pigment epithelial cells and endothelial cells on double collagen-coated honeycomb films [J].Acta Biomater,2020,101:327-343.
[52] CHANG Y H,KUMAR V B,WEN Y T,HUANG C Y,TSAI R K,DING D C.Induction of human umbilical mesenchymal stem cell differentiation into retinal pigment epithelial cells using a transwell-based co-culture system [J].Cell Transplant,2022,31:9636897221085901.
[53] ABOUTALEB KADKHODAEIAN H,SALATI A,LASHAY A.High efficient differentiation of human adipose-derived stem cells into retinal pigment epithelium-like cells in medium containing small molecules inducers with a simple method [J].Tissue Cell,2019,56:52-59.
[54] HUANG C,ZHANG J,AO M,LI Y,ZHANG C,XU Y,et al.Combination of retinal pigment epithelium cell-conditioned medium and photoreceptor outer segments stimulate mesenchymal stem cell differentiation toward a functional retinal pigment epithelium cell phenotype [J].J Cell Biochem,2012,113(2):590-598.
[55] GONG L,WU Q,SONG B,LU B,ZHANG Y.Differentiation of rat mesenchymal stem cells transplanted into the subretinal space of sodium iodate-injected rats [J].Clin Exp Ophthalmol,2008,36(7):666-671.
[56] JIANG Y,ZHANG Y,ZHANG L,WANG M,ZHANG X,LI X.Therapeutic effect of bone marrow mesenchymal stem cells on laser-induced retinal injury in mice [J].Int J Mol Sci,2014,15(6):9372-9385.
[57] FROEN R C,JOHNSEN E O,PETROVSKI G,BERENYI E,FACSKO A,BERTA A,et al.Pigment epithelial cells isolated from human peripheral iridectomies have limited properties of retinal stem cells [J].Acta Ophthalmol,2011,89(8):e635-644.
[58] AISENBREY S,LAFAUT B A,SZURMAN P,HILGERS R D,ESSER P,WALTER P,et al.Iris pigment epithelial translocation in the treatment of exudative macular degeneration:a 3-year follow-up [J].Arch Ophthalmol,2006,124(2):183-188.
[59] GAGLIARDI G,BEN M’BAREK K,GOUREAU O.Photoreceptor cell replacement in macular degeneration and retinitis pigmentosa:a pluripotent stem cell-based approach [J].Prog Retin Eye Res,2019,71:1-25.
[60] ZERTI D,HILGEN G,DORGAU B,COLLIN J,ADER M,ARMSTRONG L,et al.Transplanted pluripotent stem cell-derived photoreceptor precursors elicit conventional and unusual light responses in mice with advanced retinal degeneration [J].Stem Cells,2021,39(7):882-896.
[61] HOMMA K,OKAMOTO S,MANDAI M,GOTOH N,RAJASIMHA H K,CHANG Y S,et al.Developing rods transplanted into the degenerating retina of Crx-knockout mice exhibit neural activity similar to native photoreceptors [J].Stem Cells,2013,31(6):1149-1159.
[62] LI B,JIANG H,LI H,ZHANG B,SLAUGHTER M,YAN Z,et al.Direct conversion of adult human retinal pigmented epithelium cells to neurons with photoreceptor properties [J].Exp Biol Med (Maywood),2021,246(2):240-248.
[63] DING S L S,KOH A E,KUMAR S,ALI KHAN M S,ALZAHRANI B,MOK P L.Genetically-modified human mesenchymal stem cells to express erythropoietin enhances differentiation into retinal photoreceptors:an in-vitro study [J].J Photochem Photobiol B,2019,195:33-38.
[64] PAN T,SHEN H,YUAN S,LU G,ZHANG Y,WANG H,et al.Combined transplantation with human mesenchymal stem cells improves retinal rescue effect of human fetal RPE cells in retinal degeneration mouse model [J].Invest Ophthalmol Vis Sci,2020,61(8):9.
[65] SHIN J Y,MA D,LIM M S,CHO M S,KIM Y J,YU H G.Embryonic stem cell-derived photoreceptor precursor cells differentiated by coculture with RPE cells [J].Mol Vis,2021,27:288-299.
[66] ASSAWACHANANONT J,MANDAI M,OKAMOTO S,YAMADA C,EIRAKU M,YONEMURA S,et al.Transplantation of embryonic and induced pluripotent stem cell-derived 3D retinal sheets into retinal degenerative mice [J].Stem Cell Reports,2014,2(5):662-674.
[67] LEE I K,LUDWIG A L,PHILLIPS M J,LEE J,XIE R,SAJDAK B S,et al.Ultrathin micromolded 3D scaffolds for high-density photoreceptor layer reconstruction [J].Sci Adv,2021,7(17):eabf0344.