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Updated: Jun 15, 2025

In Vivo Modeling of the Morbid Human Genome using Danio rerio
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Published on: August 24, 2013

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评估基因治疗的精准医学方法,用于患者特异的Bietti晶体缩细胞模型

Yao Li1, Richard R Yang2, Yong-Shi Li1

  • 1Jonas Children's Vision Care, Department of Ophthalmology, Columbia University, New York, New York, USA.

JCI insight
|August 22, 2024
PubMed
概括

患者特异性诱导的多能干细胞衍生 (iPSC衍生) 视网膜色素表皮细胞显示出对治疗Bietti晶体缩症 (BCD) 的希望. 腺相关病毒介导的基因疗法有效地减少了这些BCD细胞的光诱导损伤.

关键词:
基因治疗是一种基因疗法.遗传性疾病是一种遗传性疾病.眼科医生 眼科 眼科治疗方法 治疗方法iPS 细胞 细胞 iPS 细胞

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