治疗老年性黄斑变性的基因、细胞和抗体疗法。

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
{"title":"治疗老年性黄斑变性的基因、细胞和抗体疗法。","authors":"Engin Akyol, Andrew Lotery","doi":"10.2147/BTT.S252581","DOIUrl":null,"url":null,"abstract":"<p><p>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.</p>","PeriodicalId":9025,"journal":{"name":"Biologics : Targets & Therapy","volume":null,"pages":null},"PeriodicalIF":5.3000,"publicationDate":"2020-09-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/40/8e/btt-14-83.PMC7494004.pdf","citationCount":"0","resultStr":"{\"title\":\"Gene, Cell and Antibody-Based Therapies for the Treatment of Age-Related Macular Degeneration.\",\"authors\":\"Engin Akyol, Andrew Lotery\",\"doi\":\"10.2147/BTT.S252581\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>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.</p>\",\"PeriodicalId\":9025,\"journal\":{\"name\":\"Biologics : Targets & Therapy\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":5.3000,\"publicationDate\":\"2020-09-11\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/40/8e/btt-14-83.PMC7494004.pdf\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Biologics : Targets & Therapy\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.2147/BTT.S252581\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2020/1/1 0:00:00\",\"PubModel\":\"eCollection\",\"JCR\":\"Q1\",\"JCRName\":\"MEDICINE, RESEARCH & EXPERIMENTAL\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Biologics : Targets & Therapy","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.2147/BTT.S252581","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2020/1/1 0:00:00","PubModel":"eCollection","JCR":"Q1","JCRName":"MEDICINE, RESEARCH & EXPERIMENTAL","Score":null,"Total":0}
引用次数: 0

摘要

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。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

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

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

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.

求助全文
通过发布文献求助,成功后即可免费获取论文全文。 去求助
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
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
copy
已复制链接
快去分享给好友吧!
我知道了
右上角分享
点击右上角分享
0
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术官方微信