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Pluripotent stem cell-derived retinal organoids for disease modeling and development of therapies
STEM CELLS ( IF 5.2 ) Pub Date : 2020-06-07 , DOI: 10.1002/stem.3239
Kamil Kruczek 1 , Anand Swaroop 1
Affiliation  

Retinal diseases constitute a genetically and phenotypically diverse group of clinical conditions leading to vision impairment or blindness with limited treatment options. Advances in reprogramming of somatic cells to induced pluripotent stem cells and generation of three‐dimensional organoids resembling the native retina offer promising tools to interrogate disease mechanisms and evaluate potential therapies for currently incurable retinal neurodegeneration. Next‐generation sequencing, single‐cell analysis, advanced electrophysiology, and high‐throughput screening approaches are expected to greatly expand the utility of stem cell‐derived retinal cells and organoids for developing personalized treatments. In this review, we discuss the current status and future potential of combining retinal organoids as human models with recent technologies to advance the development of gene, cell, and drug therapies for retinopathies.

中文翻译:

多能干细胞衍生的视网膜类器官用于疾病建模和治疗开发

视网膜疾病构成一组遗传和表型不同的临床病症,导致视力障碍或失明,治疗选择有限。在将体细胞重编程为诱导多能干细胞和生成类似于天然视网膜的三维类器官方面取得的进展为探究疾病机制和评估目前无法治愈的视网膜神经变性的潜在疗法提供了有希望的工具。下一代测序、单细胞分析、先进的电生理学和高通量筛选方法有望极大地扩展干细胞衍生的视网膜细胞和类器官在开发个性化治疗方面的应用。在本次审查中,
更新日期:2020-06-07
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