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：

美国国家卫生研究院; 日本学术振兴会;

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
Ader, Marius
Tanaka, Elly M.
[J]. CURRENT OPINION IN CELL BIOLOGY, 2014, 31 : 23 - 28
[2] From Progenitors to Differentiated Cells in the Vertebrate Retina
Agathocleous, Michalis
Harris, William A.
[J]. 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
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
[J]. 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
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.
[J]. NEW ENGLAND JOURNAL OF MEDICINE, 2015, 372 (20) : 1887 - 1897
[5] Cell fate determination in the vertebrate retina
Bassett, Erin A.
Wallace, Valerie A.
[J]. 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
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.
[J]. 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
Bermingham-McDonogh, Olivia
Reh, Thomas A.
[J]. NEURON, 2011, 71 (03) : 389 - 405
[8] Using human induced pluripotent stem cells to treat retinal disease
Borooah, S.
Phillips, M. J.
Bilican, B.
Wright, A. F.
Wilmut, I.
Chandran, S.
Gamm, D.
Dhillon, B.
[J]. 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
Braam, Stefan R.
Nauw, Roy
Ward-van Oostwaard, Dorien
Mummery, Christine
Passier, Robert
[J]. ANALYSIS OF CARDIAC DEVELOPMENT: FROM EMBRYO TO OLD AGE, 2010, 1188 : 52 - 57
[10] Brooks MJ, 2011, MOL VIS, V17, P3034

← 1 2 3 4 5 6 7 →