[1]万光明,余川.重视不同来源移植细胞在视网膜退行性疾病中的替代治疗作用[J].眼科新进展,2022,42(8):589-596.[doi:10.13389/j.cnki.rao.2022.0121]
 WAN Guangming,YU Chuan.Effect of the replacement therapy of transplanted cells from different sources in retinal degenerative diseases[J].Recent Advances in Ophthalmology,2022,42(8):589-596.[doi:10.13389/j.cnki.rao.2022.0121]
点击复制

重视不同来源移植细胞在视网膜退行性疾病中的替代治疗作用/HTML
分享到:

《眼科新进展》[ISSN:1003-5141/CN:41-1105/R]

卷:
42卷
期数:
2022年8期
页码:
589-596
栏目:
述评
出版日期:
2022-08-06

文章信息/Info

Title:
Effect of the replacement therapy of transplanted cells from different sources in retinal degenerative diseases
作者:
万光明余川
450052 河南省郑州市,郑州大学第一附属医院眼科
Author(s):
WAN GuangmingYU Chuan
Department of Ophthalmology,the First Affiliated Hospital of Zhengzhou University,Zhengzhou 450052,Henan Province,China
关键词:
视网膜退行性疾病干细胞视网膜细胞细胞移植替代治疗
Keywords:
retinal degenerative diseases stem cells retinal cells cell transplantation replacement therapy
分类号:
R774.1
DOI:
10.13389/j.cnki.rao.2022.0121
文献标志码:
A
摘要:
视网膜退行性疾病是导致不可逆性视力损害和失明的主要原因之一,尚无有效的治疗方法。细胞疗法因具有替代受损或丧失功能细胞的作用,在视网膜退行性疾病的再生医学中受到广泛关注。不同来源移植细胞的替代治疗在基础研究和临床试验中显示了一定的安全性和有效性,但仍存在伦理、免疫排斥、致瘤性等不足。病变区微环境也影响着移植细胞的存活,使细胞替代治疗的临床应用受到阻碍。随着移植细胞种类增加和移植技术更新,通过重编程、基因修饰、三维培养和生物支架等的应用,视网膜退行性疾病的细胞替代治疗效果得到不断优化。不同学科间交叉合作有效解决了不同来源移植细胞的一些潜在问题,在细胞悬液、细胞膜片和视网膜类器官替代治疗视网膜退行性疾病方面取得显著进展。
Abstract:
Retinal degenerative disease is one of the leading causes of irreversible visual impairment and blindness, and there is no effective treatment for it. Recently, cell therapy has attracted widespread attention in regenerative medicine for retinal degenerative diseases due to its ability to replace damaged or dysfunctional cells. The replacement therapy of transplanted cells from different sources has shown certain safety and effectiveness in basic research and clinical trials, but there are still some deficiencies, such as ethics, immune rejection, and tumorigenicity. Besides, the survival of transplanted cells is susceptible to the microenvironment of the lesion, hindering the clinical application of cell replacement therapy. With the increase of transplanted cells and upgrade of transplantation technology, the outcome of cell replacement therapy for retinal degenerative diseases has been continuously optimized through the application of reprogramming, gene modification, three-dimensional culture, and biological scaffolding. The interdisciplinary cooperation offers an effective solution to some potential problems caused by transplanted cells from different sources, and remarkable progress has been made in cell suspension, cell sheet, and retinal organoid replacement therapy for retinal degenerative diseases.

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] SPTER T,ASSUNO 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.

相似文献/References:

[1]王娜 刘振山 冯云 陈银银 薛志刚 范国平.角膜内皮细胞体外培养的研究进展[J].眼科新进展,2013,33(6):000.
[2]冯云 刘振山 王娜 薛志刚 范国平.体外神经视网膜重构及退行性眼病的临床治疗展望[J].眼科新进展,2013,33(8):000.
[3]胡雅敏 罗敏 谷平.干细胞在视网膜损伤修复中的应用进展[J].眼科新进展,2013,33(2):000.
[4]李筱荣.干细胞在视网膜疾病中的应用研究进展[J].眼科新进展,2013,33(10):000.
[5]高珍,彭广华.小分子化合物在干细胞诱导分化为视网膜色素上皮细胞中的应用[J].眼科新进展,2016,36(7):688.[doi:10.13389/j.cnki.rao.2016.0183]
 GAO Zhen,PENG Guang-Hua.Application of small molecule compounds in inducing differentiation of stem cells into retinal pigment epithelial cells[J].Recent Advances in Ophthalmology,2016,36(8):688.[doi:10.13389/j.cnki.rao.2016.0183]
[6]张青林,张旭.干细胞治疗青光眼的研究进展[J].眼科新进展,2017,37(9):886.[doi:10.13389/j.cnki.rao.2017.0225]
 ZHANG Qing-Lin,ZHANG Xu.Stem cells for the treatment of glaucoma[J].Recent Advances in Ophthalmology,2017,37(8):886.[doi:10.13389/j.cnki.rao.2017.0225]
[7]王霞,刘瀛,刘旭,等.诱导多功能干细胞在青光眼治疗中的应用研究进展[J].眼科新进展,2017,37(12):1185.[doi:10.13389/j.cnki.rao.2017.0299]
 WANG Xia,LIU Ying,LIU Xu,et al.Recent advances of induced pluripotent stem cells in glaucoma treatment[J].Recent Advances in Ophthalmology,2017,37(8):1185.[doi:10.13389/j.cnki.rao.2017.0299]
[8]于莎莎,赵云.干细胞对视神经损伤的修复作用[J].眼科新进展,2018,38(3):298.[doi:10.13389/j.cnki.rao.2018.0070]
 YU Sha-Sha,ZHAO Yun.Latest progress in stem cell transplantation for the treatment of optic nerve injury[J].Recent Advances in Ophthalmology,2018,38(8):298.[doi:10.13389/j.cnki.rao.2018.0070]
[9]李润璞,金鑫.基因组编辑技术在遗传性视网膜营养不良基因治疗和细胞治疗中的应用[J].眼科新进展,2020,40(3):290.[doi:10.13389/j.cnki.rao.2020.0068]
 LI Runpu,JIN Xin.Application of genomic editing technology in gene therapy and cell therapy of inherited retinal dystrophies[J].Recent Advances in Ophthalmology,2020,40(8):290.[doi:10.13389/j.cnki.rao.2020.0068]
[10]舒会叶,韦芊,邵毅.视网膜类器官3D培养技术的研究进展[J].眼科新进展,2022,42(10):824.[doi:10.13389/j.cnki.rao.2022.0170]
 SHU Huiye,WEI Qian,SHAO Yi.Advances in the study of 3D culture technology for retinal organoids[J].Recent Advances in Ophthalmology,2022,42(8):824.[doi:10.13389/j.cnki.rao.2022.0170]

备注/Memo

备注/Memo:
国家重点研发计划项目(编号:2018YFA0107300)
更新日期/Last Update: 2022-08-05