Patient iPSC-derived retinal organoids: Observable retinal diseases in-a-dish

被引:8
作者
Zhang, Xiao-Hui [1 ]
Jin, Zi-Bing [1 ]
机构
[1] Capital Med Univ, Beijing Inst Ophthalmol, Beijing Tongren Eye Ctr, Beijing Tongren Hosp,Beijing Ophthalmol & Visual, Beijing, Peoples R China
基金
国家重点研发计划; 北京市自然科学基金;
关键词
Induced pluripotent stem cells; Retinal organoids; Inherited retinal dystrophy; Gene editing; Disease modeling; PLURIPOTENT STEM-CELLS; RETINITIS-PIGMENTOSA; GENE; REVEALS; RETINOBLASTOMA; GENERATION; MUTATIONS; CORRECTS;
D O I
10.14670/HH-18-307
中图分类号
Q2 [细胞生物学];
学科分类号
071009 ; 090102 ;
摘要
Induced pluripotent stem cells (iPSCs), reprogrammed from human somatic cells, hold the capacity to differentiate into most human body cells. iPSCs can be differentiated into retinal organoids, a three-dimensional structured retina containing various retinal cells. Patient-specific retinal organoids provide a powerful disease model to recapitulate the disease to study the pathogenesis of inherited retinal dystrophies, to screen or discover new drugs, and most importantly to supply an unlimited cell source for retinal regeneration.
引用
收藏
页码:705 / 710
页数:6
相关论文
共 38 条
  • [1] Merging organoid and organ-on-a-chip technology to generate complex multi-layer tissue models in a human retina-on-a-chip platform
    Achberger, Kevin
    Probst, Christopher
    Haderspeck, Jasmin
    Bolz, Sylvia
    Rogal, Julia
    Chuchuy, Johanna
    Nikolova, Marina
    Cora, Virginia
    Antkowiak, Lena
    Haq, Wadood
    Shen, Nian
    Schenke-Layland, Katja
    Ueffing, Marius
    Liebau, Stefan
    Loskill, Peter
    [J]. ELIFE, 2019, 8
  • [2] Stem cell-based retina models
    Achberger, Kevin
    Haderspeck, Jasmin C.
    Kleger, Alexander
    Liebau, Stefan
    [J]. ADVANCED DRUG DELIVERY REVIEWS, 2019, 140 : 33 - 50
  • [3] Generation of gene-corrected human induced pluripotent stem cell lines derived from retinitis pigmentosa patient with Ser331Cysfs*5 mutation in MERTK
    Artero Castro, Ana
    Long, Kathleen
    Bassett, Andrew
    Machuca, Candela
    Leon, Marian
    Avila-Fernandez, Almudena
    Corton, Marta
    Vidal-Puig, Toni
    Ayuso, Carmen
    Lukovic, Dunja
    Erceg, Slaven
    [J]. STEM CELL RESEARCH, 2019, 34
  • [4] Pluripotent stem cells in disease modelling and drug discovery
    Avior, Yishai
    Sagi, Ido
    Benvenisty, Nissim
    [J]. NATURE REVIEWS MOLECULAR CELL BIOLOGY, 2016, 17 (03) : 170 - 182
  • [5] In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa
    Bakondi, Benjamin
    Lv, Wenjian
    Lui, Bin
    Jones, Melissa K.
    Tsai, Yuchun
    Kim, Kevin J.
    Levy, Rachelle
    Akhtar, Aslam Abbasi
    Breunig, Joshua J.
    Svendseni, Clive N.
    Wang, Shaomei
    [J]. MOLECULAR THERAPY, 2016, 24 (03) : 556 - 563
  • [6] Precision Medicine: Genetic Repair of Retinitis Pigmentosa in Patient-Derived Stem Cells
    Bassuk, Alexander G.
    Zheng, Andrew
    Li, Yao
    Tsang, Stephen H.
    Mahajan, Vinit B.
    [J]. SCIENTIFIC REPORTS, 2016, 6
  • [7] Using CRISPR-Cas9 to Generate Gene-Corrected Autologous iPSCs for the Treatment of Inherited Retinal Degeneration
    Burnight, Erin R.
    Gupta, Manav
    Wiley, Luke A.
    Anfinson, Kristin R.
    Tran, Audrey
    Triboulet, Robinson
    Hoffmann, Jeremy M.
    Klaahsen, Darcey L.
    Andorf, Jeaneen L.
    Jiao, Chunhua
    Sohn, Elliott H.
    Adur, Malavika K.
    Ross, Jason W.
    Mullins, Robert F.
    Daley, George Q.
    Schlaeger, Thorsten M.
    Stone, Edwin M.
    Tucker, Budd A.
    [J]. MOLECULAR THERAPY, 2017, 25 (09) : 1999 - 2013
  • [8] Disrupted alternative splicing for genes implicated in splicing and ciliogenesis causes PRPF31 retinitis pigmentosa
    Buskin, Adriana
    Zhu, Lili
    Chichagova, Valeria
    Basu, Basudha
    Mozaffari-Jovin, Sina
    Dolan, David
    Droop, Alastair
    Collin, Joseph
    Bronstein, Revital
    Mehrotra, Sudeep
    Farkas, Michael
    Hilgen, Gerrit
    White, Kathryn
    Pan, Kuan-Ting
    Treumann, Achim
    Hallam, Dean
    Bialas, Katarzyna
    Chung, Git
    Mellough, Carla
    Ding, Yuchun
    Krasnogor, Natalio
    Przyborski, Stefan
    Zwolinski, Simon
    Al-Aama, Jumana
    Alharthi, Sameer
    Xu, Yaobo
    Wheway, Gabrielle
    Szymanska, Katarzyna
    McKibbin, Martin
    Inglehearn, Chris F.
    Elliott, David J.
    Lindsay, Susan
    Ali, Robin R.
    Steel, David H.
    Armstrong, Lyle
    Sernagor, Evelyne
    Urlaub, Henning
    Pierce, Eric
    Luehrmann, Reinhard
    Grellscheid, Sushma-Nagaraja
    Johnson, Colin A.
    Lako, Majlinda
    [J]. NATURE COMMUNICATIONS, 2018, 9
  • [9] Gene Correction Reverses Ciliopathy and Photoreceptor Loss in iPSC-Derived Retinal Organoids from Retinitis Pigmentosa Patients
    Deng, Wen-Li
    Gao, Mei-Ling
    Lei, Xin-Lan
    Lv, Ji-Neng
    Zhao, Huan
    He, Kai-Wen
    Xia, Xi-Xi
    Li, Ling-Yun
    Chen, Yu-Chen
    Li, Yan-Ping
    Pan, Deng
    Xue, Tian
    Jin, Zi-Bing
    [J]. STEM CELL REPORTS, 2018, 10 (04): : 1267 - 1281
  • [10] Retinoblastoma
    Dimaras, Helen
    Kimani, Kahaki
    Dimba, Elizabeth A. O.
    Gronsdahl, Peggy
    White, Abby
    Chan, Helen S. L.
    Gallie, Brenda L.
    [J]. LANCET, 2012, 379 (9824) : 1436 - 1446