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

[31] Organogenesis in a dish: Modeling development and disease using organoid technologies [J].

Lancaster, Madeline A. ;

Knoblich, Juergen A. .

SCIENCE, 2014, 345 (6194)

[32] Large-scale screening using familial dysautonomia induced pluripotent stem cells identifies compounds that rescue IKBKAP expression [J].

Lee, Gabsang ;

Ramirez, Christina N. ;

Kim, Hyesoo ;

Zeltner, Nadja ;

Liu, Becky ;

Radu, Constantin ;

Bhinder, Bhavneet ;

Kim, Yong Jun ;

Choi, In Young ;

Mukherjee-Clavin, Bipasha ;

Djaballah, Hakim ;

Studer, Lorenz .

NATURE BIOTECHNOLOGY, 2012, 30 (12) :1244-+

[33] Retinal repair by transplantation of photoreceptor precursors [J].

MacLaren, R. E. ;

Pearson, R. A. ;

MacNeil, A. ;

Douglas, R. H. ;

Salt, T. E. ;

Akimoto, M. ;

Swaroop, A. ;

Sowden, J. C. ;

Ali, R. R. .

NATURE, 2006, 444 (7116) :203-207

[34] RNA-Guided Human Genome Engineering via Cas9 [J].

Mali, Prashant ;

Yang, Luhan ;

Esvelt, Kevin M. ;

Aach, John ;

Guell, Marc ;

DiCarlo, James E. ;

Norville, Julie E. ;

Church, George M. .

SCIENCE, 2013, 339 (6121) :823-826

[35] IGF-1 Signaling Plays an Important Role in the Formation of Three-Dimensional Laminated Neural Retina and Other Ocular Structures From Human Embryonic Stem Cells [J].

Mellough, Carla B. ;

Collin, Joseph ;

Khazim, Mahmoud ;

White, Kathryn ;

Sernagor, Evelyne ;

Steel, David H. W. ;

Lako, Majlinda .

STEM CELLS, 2015, 33 (08) :2416-2430

[36] Optic Vesicle-like Structures Derived from Human Pluripotent Stem Cells Facilitate a Customized Approach to Retinal Disease Treatment [J].

Meyer, Jason S. ;

Howden, Sara E. ;

Wallace, Kyle A. ;

Verhoeven, Amelia D. ;

Wright, Lynda S. ;

Capowski, Elizabeth E. ;

Pinilla, Isabel ;

Martin, Jessica M. ;

Tian, Shulan ;

Stewart, Ron ;

Pattnaik, Bikash ;

Thomson, James A. ;

Gamm, David M. .

STEM CELLS, 2011, 29 (08) :1206-1218

[37] Modeling early retinal development with human embryonic and induced pluripotent stem cells [J].

Meyer, Jason S. ;

Shearer, Rebecca L. ;

Capowski, Elizabeth E. ;

Wright, Lynda S. ;

Wallace, Kyle A. ;

McMillan, Erin L. ;

Zhang, Su-Chun ;

Gamm, David M. .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2009, 106 (39) :16698-16703

[38] An Essential Role for RAX Homeoprotein and NOTCH-HES Signaling in Otx2 Expression in Embryonic Retinal Photoreceptor Cell Fate Determination [J].

Muranishi, Yuki ;

Terada, Koji ;

Inoue, Tatsuya ;

Katoh, Kimiko ;

Tsujii, Toshinori ;

Sanuki, Rikako ;

Kurokawa, Daisuke ;

Aizawa, Shinichi ;

Tamaki, Yasuhiro ;

Furukawa, Takahisa .

JOURNAL OF NEUROSCIENCE, 2011, 31 (46) :16792-16807

[39] Self-Formation of Optic Cups and Storable Stratified Neural Retina from Human ESCs [J].

Nakano, Tokushige ;

Ando, Satoshi ;

Takata, Nozomu ;

Kawada, Masako ;

Muguruma, Keiko ;

Sekiguchi, Kiyotoshi ;

Saito, Koichi ;

Yonemura, Shigenobu ;

Eiraku, Mototsugu ;

Sasai, Yoshiki .

CELL STEM CELL, 2012, 10 (06) :771-785

[40] Long-Term, Stable Differentiation of Human Embryonic Stem Cell-Derived Neural Precursors Grafted into the Adult Mammalian Neostriatum [J].

Nasonkin, Igor ;

Mahairaki, Vasiliki ;

Xu, Leyan ;

Hatfield, Glen ;

Cummings, Brian J. ;

Eberhart, Charles ;

Ryugo, David K. ;

Maric, Dragan ;

Bar, Eli ;

Koliatsos, Vassilis E. .

STEM CELLS, 2009, 27 (10) :2414-2426

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