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用于治疗年龄相关性黄斑变性的基因、细胞和抗体疗法。

Gene, Cell and Antibody-Based Therapies for the Treatment of Age-Related Macular Degeneration.

作者信息

Akyol Engin, Lotery Andrew

机构信息

Clinical Neurosciences Research Group, Faculty of Medicine, University of Southampton, Southampton General Hospital, Southampton SO16 6YD, UK.

出版信息

Biologics. 2020 Sep 11;14:83-94. doi: 10.2147/BTT.S252581. eCollection 2020.

DOI:10.2147/BTT.S252581
PMID:32982165
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC7494004/
Abstract

Here we discuss antibody, cell and gene-based therapies that are currently available and under investigation for both wet and dry age-related macular degeneration (AMD). We initially discuss ocular anatomy, AMD modelling as well as the underlying pathophysiology of AMD. The antibody-based trials which have revolutionised the management of wet AMD are reviewed. The latest concepts in antibody therapy for wet AMD such as the port delivery systems, bispecific antibodies, designed ankyrin repeat protein (DARPINs) and brolucizumab are explored. Furthermore, the antibody-based trials targeting the complement pathway to reduce progression of geographic atrophy (GA) in dry AMD are discussed. Stem cell therapy and gene therapy are novel treatment modalities with no established clinical use in wet or dry AMD. Here, we discuss their efficacy so far in clinical trials. Their benefits and risk in the treatment of both wet and dry AMD are evaluated.

摘要

在此,我们讨论目前可用于湿性和干性年龄相关性黄斑变性(AMD)以及正在研究中的基于抗体、细胞和基因的疗法。我们首先讨论眼部解剖结构、AMD模型以及AMD的潜在病理生理学。对彻底改变湿性AMD治疗管理的基于抗体的试验进行了综述。探讨了湿性AMD抗体治疗的最新概念,如端口递送系统、双特异性抗体、设计锚蛋白重复蛋白(DARPINs)和布罗珠单抗。此外,还讨论了针对补体途径以减少干性AMD中地图样萎缩(GA)进展的基于抗体的试验。干细胞疗法和基因疗法是新型治疗方式,在湿性或干性AMD中尚未确立临床应用。在此,我们讨论它们迄今为止在临床试验中的疗效。评估了它们在治疗湿性和干性AMD中的益处和风险。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/f286/7494004/abcaab18259c/BTT-14-83-g0001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/f286/7494004/abcaab18259c/BTT-14-83-g0001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/f286/7494004/abcaab18259c/BTT-14-83-g0001.jpg

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