Patient-Specific iPSC-Derived RPE for Modeling of Retinal Diseases.
Patient-Specific iPSC-Derived RPE for Modeling of Retinal Diseases.
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DOI：
10.3390/jcm4040567
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发表时间：
2015-03-31
影响因子：
3.9
通讯作者：
Tsang SH
中科院分区：
文献类型：
--
作者：
Nguyen HV;Li Y;Tsang SH
遗传性视网膜疾病，如年龄相关性黄斑变性和视网膜色素变性，是发达国家失明的主要原因。目前，这些疾病的治疗方法有限。最近，人们对使用患者特异性诱导多能干细胞（iPSC）作为这些病症的治疗的可能性给予了相当大的关注。从成体体细胞重编程的iPSC提供了在体外产生患者特异性细胞系的可能性。在这篇综述中，我们将讨论有关视网膜疾病的iPSC建模，iPSC衍生的视网膜色素上皮细胞（RPE）细胞的基因治疗和视网膜移植的当前文献。我们将专注于使用从遗传性眼病患者中产生的iPSC来测试基因或药物治疗的疗效，阐明以前未知的疾病机制和途径，并作为细胞替代的自体细胞来源。
Inherited retinal diseases, such as age-related macular degeneration and retinitis pigmentosa, are the leading cause of blindness in the developed world. Currently, treatments for these conditions are limited. Recently, considerable attention has been given to the possibility of using patient-specific induced pluripotent stem cells (iPSCs) as a treatment for these conditions. iPSCs reprogrammed from adult somatic cells offer the possibility of generating patient-specific cell lines in vitro. In this review, we will discuss the current literature pertaining to iPSC modeling of retinal disease, gene therapy of iPSC-derived retinal pigmented epithelium (RPE) cells, and retinal transplantation. We will focus on the use of iPSCs created from patients with inherited eye diseases for testing the efficacy of gene or drug-based therapies, elucidating previously unknown mechanisms and pathways of disease, and as a source of autologous cells for cell replacement.
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影响因子：
4.4
作者：
Phillips, M. Joseph;Wallace, Kyle A.;Gamm, David M.
通讯作者：
Gamm, David M.
影响因子：
3.7
作者：
Jin ZB;Okamoto S;Osakada F;Homma K;Assawachananont J;Hirami Y;Iwata T;Takahashi M
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Takahashi M
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158.5
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23.9
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Kim D;Kim CH;Moon JI;Chung YG;Chang MY;Han BS;Ko S;Yang E;Cha KY;Lanza R;Kim KS
通讯作者：
Kim KS
DOI：
10.1126/science.1232033
发表时间：
2013-02-15
期刊：
Science (New York, N.Y.)
影响因子：
--
作者：
Mali P;Yang L;Esvelt KM;Aach J;Guell M;DiCarlo JE;Norville JE;Church GM
通讯作者：
Church GM