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

IF 5.3 Q1 MEDICINE, RESEARCH & EXPERIMENTAL Biologics : Targets & Therapy Pub Date : 2020-09-11 eCollection Date: 2020-01-01 DOI:10.2147/BTT.S252581
Engin Akyol, Andrew Lotery
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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.

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治疗老年性黄斑变性的基因、细胞和抗体疗法。
在此,我们将讨论目前可用于治疗湿性和干性老年性黄斑变性(AMD)的抗体、细胞和基因疗法。我们首先讨论了眼部解剖、AMD 模型以及 AMD 的基本病理生理学。我们还回顾了彻底改变湿性黄斑变性治疗的抗体试验。探讨了湿性老年黄斑变性的抗体疗法的最新概念,如端口传输系统、双特异性抗体、设计的ankyrin重复蛋白(DARPINs)和brolucizumab。此外,还讨论了针对补体途径的抗体试验,以减少干性AMD中地理萎缩(GA)的进展。干细胞疗法和基因疗法是新型的治疗方式,在湿性或干性AMD中尚未得到成熟的临床应用。在此,我们将讨论它们迄今为止在临床试验中的疗效。我们还评估了干细胞疗法和基因疗法在治疗湿性和干性老年性黄斑变性中的益处和风险。
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来源期刊
Biologics : Targets & Therapy
Biologics : Targets & Therapy MEDICINE, RESEARCH & EXPERIMENTAL-
CiteScore
8.30
自引率
0.00%
发文量
22
审稿时长
16 weeks
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