Gene and cell therapy for age-related macular degeneration: A review

IF 5.1 2区 医学 Q1 OPHTHALMOLOGY
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引用次数: 0

Abstract

Age-related macular degeneration (AMD) is the most common cause of irreversible vision loss among the elderly in Western communities, with an estimated global prevalence of 10 – 20% in people older than 65 years. AMD leads to central vision loss due to degeneration of the photoreceptors, retinal pigment epithelium and the choriocapillaris. Beckman’s classification for AMD, based upon color fundus photographs, divides the disease into early, intermediate, and late forms. The late, vision-threatening stage includes both neovascular AMD and geographic atrophy. Despite its high prevalence and impact on patients’ quality of life, treatment options for AMD are limited. While neovascular AMD can be medically managed with anti-VEGF intravitreal injections, until very recently there has been no approved treatment options for atrophic AMD; however, in February 2023 the first treatment for geographic atrophy – pegcetacoplan – was approved by the US FDA. We describe the current landscape of potential gene and cell therapeutic strategies for late-stage AMD, with an emphasis on the therapeutic options that might become available in the next few years.

老年性黄斑变性的基因和细胞疗法:综述。
年龄相关性黄斑变性(AMD)是西方社区老年人不可逆转的视力丧失的最常见原因,估计全球 65 岁以上老年人的发病率为 10%-20%。由于光感受器、视网膜色素上皮和绒毛膜变性,黄斑变性导致中心视力丧失。贝克曼根据彩色眼底照片对老年性视网膜病变进行了分类,将这种疾病分为早期、中期和晚期。晚期威胁视力,包括新生血管性黄斑变性和地理萎缩。尽管老年性黄斑变性的发病率很高,对患者的生活质量也有影响,但治疗方法却很有限。虽然新生血管性黄斑变性可以通过抗血管内皮生长因子(anti-VEGF)玻璃体内注射进行治疗,但直到最近,萎缩性黄斑变性的治疗方案还没有获得批准;不过,2023 年 2 月,美国 FDA 批准了第一种治疗地理萎缩的药物--培西他克普兰(pegcetacoplan)。我们介绍了目前针对晚期老年性黄斑变性的潜在基因和细胞治疗策略,重点是未来几年可能出现的治疗方案。
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来源期刊
Survey of ophthalmology
Survey of ophthalmology 医学-眼科学
CiteScore
10.30
自引率
2.00%
发文量
138
审稿时长
14.8 weeks
期刊介绍: Survey of Ophthalmology is a clinically oriented review journal designed to keep ophthalmologists up to date. Comprehensive major review articles, written by experts and stringently refereed, integrate the literature on subjects selected for their clinical importance. Survey also includes feature articles, section reviews, book reviews, and abstracts.
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