开发自体诱导多能干细胞(iPSC)衍生视网膜色素上皮(RPE)替代疗法的考虑因素。

IF 7.8 2区 医学 Q1 MEDICINE, RESEARCH & EXPERIMENTAL Cold Spring Harbor perspectives in medicine Pub Date : 2024-03-01 DOI:10.1101/cshperspect.a041295
Devika Bose, Davide Ortolan, Mitra Farnoodian, Ruchi Sharma, Kapil Bharti
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引用次数: 0

摘要

细胞替代疗法是一种新型疗法,包括诱导多能干细胞(iPSC)衍生的组织,旨在替代退化细胞。细胞替代治疗视网膜变性疾病是细胞疗法的最前沿,因为眼球可用于外科手术,而且视网膜变性疾病的巨大医疗需求尚未得到满足,目前尚无治疗方案。世界各地正在利用干细胞衍生的视网膜色素上皮(RPE)进行临床试验。本综述重点介绍开发 iPSC 衍生 RPE 细胞疗法时的科学和监管考虑因素,包括制定稳健高效的分化方案、细胞验证的关键质量控制检测、选择合适的动物模型进行临床前测试,以及决定最终批准进行首次人体临床试验的监管方面。
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Considerations for Developing an Autologous Induced Pluripotent Stem Cell (iPSC)-Derived Retinal Pigment Epithelium (RPE) Replacement Therapy.

Cell-replacement therapies are a new class of treatments, which include induced pluripotent stem cell (iPSC)-derived tissues that aim to replace degenerated cells. iPSCs can potentially be used to generate any cell type of the body, making them a powerful tool for treating degenerative diseases. Cell replacement for retinal degenerative diseases is at the forefront of cell therapies, given the accessibility of the eye for surgical procedures and a huge unmet medical need for retinal degenerative diseases with no current treatment options. Clinical trials are ongoing in different parts of the world using stem cell-derived retinal pigment epithelium (RPE). This review focuses on scientific and regulatory considerations when developing an iPSC-derived RPE cell therapy from the development of a robust and efficient differentiation protocol to critical quality control assays for cell validation, the choice of an appropriate animal model for preclinical testing, and the regulatory aspects that dictate the final approval for proceeding to a first-in-human clinical trial.

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来源期刊
Cold Spring Harbor perspectives in medicine
Cold Spring Harbor perspectives in medicine MEDICINE, RESEARCH & EXPERIMENTAL-
CiteScore
14.30
自引率
1.90%
发文量
44
审稿时长
4-8 weeks
期刊介绍: Cold Spring Harbor Perspectives in Medicine is a monthly online publication comprising reviews on different aspects of a variety of diseases, covering everything from the molecular and cellular bases of disease to translational medicine and new therapeutic strategies. Cold Spring Harbor Perspectives in Medicine is thus unmatched in its depth of coverage and represents an essential source where readers can find informed surveys and critical discussion of advances in molecular medicine.
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