Treatment Paradigms for Retinal and Macular Diseases Using 3-D Retina Cultures Derived From Human Reporter Pluripotent Stem Cell Lines

被引：25

作者：

Kaewkhaw, Rossukon ^{[1
,2
]}

Swaroop, Manju ^{[3
]}

Homma, Kohei ^{[1
,6
]}

Nakamura, Jutaro ^{[1
,7
]}

Brooks, Matthew ^{[1
]}

Kaya, Koray Dogan ^{[1
]}

Chaitankar, Vijender ^{[1
]}

Michael, Sam ^{[3
]}

Tawa, Gregory ^{[3
]}

Zou, Jizhong ^{[4
]}

Rao, Mahendra ^{[5
]}

Zheng, Wei ^{[3
]}

Cogliati, Tiziana ^{[1
]}

Swaroop, Anand ^{[1
]}

机构：

[1] NEI, Neurobiol Neurodegenerat & Repair Lab, NIH, Bethesda, MD 20892 USA

[2] Mahidol Univ, Ramathibodi Hosp, Fac Med, Res Ctr, Bangkok 10400, Thailand

[3] NIH, Natl Therapeut Rare & Neglected Dis, Natl Ctr Adv Translat Sci, Rockville, MD USA

[4] NHLBI, iPSC Core, Ctr Mol Med, Bldg 10, Bethesda, MD 20892 USA

[5] New York Stem Cell Fdn, Res Inst, New York, NY USA

[6] Nippon Med Sch, Dept Physiol, 1-1-5 Sendagi, Tokyo 113, Japan

[7] Yokohama City Univ, Dept Ophthalmol, Sch Med, Yokohama, Kanagawa 232, Japan

来源：

INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE | 2016年 / 57卷 / 05期

基金：

日本学术振兴会; 美国国家卫生研究院;

关键词：

human retina development; disease modeling; photoreceptor; high throughput screening; chemical screens; drug discovery; transcriptome; next generation sequencing; three-dimensional organoid culture; fluorescent reporter; HUMAN IPS CELLS; GENE-THERAPY; PHOTORECEPTOR PRECURSORS; NEURAL RETINA; REGENERATIVE MEDICINE; FATE DETERMINATION; VERTEBRATE RETINA; HUMAN ESCS; DEGENERATION; EXPRESSION;

D O I：

10.1167/iovs.15-17639

中图分类号：

R77 [眼科学];

学科分类号：

100212 ;

摘要：

We discuss the use of pluripotent stem cell lines carrying fluorescent reporters driven by retinal promoters to derive three-dimensional (3-D) retina in culture and how this system can be exploited for elucidating human retinal biology, creating disease models in a dish, and designing targeted drug screens for retinal and macular degeneration. Furthermore, we realize that stem cell investigations are labor-intensive and require extensive resources. To expedite scientific discovery by sharing of resources and to avoid duplication of efforts, we propose the formation of a Retinal Stem Cell Consortium. In the field of vision, such collaborative approaches have been enormously successful in elucidating genetic susceptibility associated with age-related macular degeneration.

引用

页码：ORSFl1 / ORSFl11

页数：11

共 68 条

[1] Modeling human development in 3D culture [J].

Ader, Marius ;

Tanaka, Elly M. .

CURRENT OPINION IN CELL BIOLOGY, 2014, 31 :23-28

[2] From Progenitors to Differentiated Cells in the Vertebrate Retina [J].

Agathocleous, Michalis ;

Harris, William A. .

ANNUAL REVIEW OF CELL AND DEVELOPMENTAL BIOLOGY, 2009, 25 :45-69

[3] Targeting of GFP to newborn rods by Nrl promoter and temporal expression profiling of flow-sorted photoreceptors [J].

Akimoto, M ;

Cheng, H ;

Zhu, DX ;

Brzezinski, JA ;

Khanna, R ;

Filippova, E ;

Oh, ECT ;

Jing, YZ ;

Linares, JL ;

Brooks, M ;

Zareparsi, S ;

Mears, AJ ;

Hero, A ;

Glaser, T ;

Swaroop, A .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2006, 103 (10) :3890-3895

[4] Long-Term Effect of Gene Therapy on Leber's Congenital Amaurosis [J].

Bainbridge, J. W. B. ;

Mehat, M. S. ;

Sundaram, V. ;

Robbie, S. J. ;

Barker, S. E. ;

Ripamonti, C. ;

Georgiadis, A. ;

Mowat, F. M. ;

Beattie, S. G. ;

Gardner, P. J. ;

Feathers, K. L. ;

Luong, V. A. ;

Yzer, S. ;

Balaggan, K. ;

Viswanathan, A. ;

de Ravel, T. J. L. ;

Casteels, I. ;

Holder, G. E. ;

Tyler, N. ;

Fitzke, F. W. ;

Weleber, R. G. ;

Nardini, M. ;

Moore, A. T. ;

Thompson, D. A. ;

Petersen-Jones, S. M. ;

Michaelides, M. ;

van den Born, L. I. ;

Stockman, A. ;

Smith, A. J. ;

Rubin, G. ;

Ali, R. R. .

NEW ENGLAND JOURNAL OF MEDICINE, 2015, 372 (20) :1887-1897

[5] Cell fate determination in the vertebrate retina [J].

Bassett, Erin A. ;

Wallace, Valerie A. .

TRENDS IN NEUROSCIENCES, 2012, 35 (09) :565-573

[6] Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa [J].

Beltran, William A. ;

Cideciyan, Artur V. ;

Lewin, Alfred S. ;

Iwabe, Simone ;

Khanna, Hemant ;

Sumaroka, Alexander ;

Chiodo, Vince A. ;

Fajardo, Diego S. ;

Roman, Alejandro J. ;

Deng, Wen-Tao ;

Swider, Malgorzata ;

Aleman, Tomas S. ;

Boye, Sanford L. ;

Genini, Sem ;

Swaroop, Anand ;

Hauswirth, William W. ;

Jacobson, Samuel G. ;

Aguirre, Gustavo D. .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2012, 109 (06) :2132-2137

[7] Regulated Reprogramming in the Regeneration of Sensory Receptor Cells [J].

Bermingham-McDonogh, Olivia ;

Reh, Thomas A. .

NEURON, 2011, 71 (03) :389-405

[8] Using human induced pluripotent stem cells to treat retinal disease [J].

Borooah, S. ;

Phillips, M. J. ;

Bilican, B. ;

Wright, A. F. ;

Wilmut, I. ;

Chandran, S. ;

Gamm, D. ;

Dhillon, B. .

PROGRESS IN RETINAL AND EYE RESEARCH, 2013, 37 :163-181

[9] Inhibition of ROCK improves survival of human embryonic stem cell-derived cardiomyocytes after dissociation [J].

Braam, Stefan R. ;

Nauw, Roy ;

Ward-van Oostwaard, Dorien ;

Mummery, Christine ;

Passier, Robert .

ANALYSIS OF CARDIAC DEVELOPMENT: FROM EMBRYO TO OLD AGE, 2010, 1188 :52-57

[10]

Brooks MJ, 2011, MOL VIS, V17, P3034

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