AMD Genomics: Non-Coding RNAs as Biomarkers and Therapeutic Targets.

AMD Genomics: Non-Coding RNAs as Biomarkers and Therapeutic Targets.
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DOI:
10.3390/jcm11061484
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发表时间:
2022-03-09
影响因子:
3.9
通讯作者:
DeAngelis MM
DeAngelis MM
中科院分区:
医学2区
文献类型:
--
作者:
Zhang C;Owen LA;Lillvis JH;Zhang SX;Kim IK;DeAngelis MM

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视网膜相关性黄斑变性(AMD)是一种进行性神经退行性疾病,是世界上老龄化人口失明的主要原因。虽然AMD的临床阶段和形式已经阐明,但需要更具体的预后工具来确定早期和中期AMD患者何时进展到AMD的晚期阶段。该领域的另一个挑战是适当开发用于中期AMD和晚期萎缩性AMD的疗法。在许多阴性临床试验之后,抗C5剂和抗C3剂最近在3期临床试验中显示出有希望的结果,在减缓地图状萎缩(AMD的晚期形式)的生长方面。有趣的是,这两种药物似乎都与湿性AMD的发病率增加有关,湿性AMD是该疾病的另一种晚期形式,并且需要频繁的玻璃体内注射。当然,仍然需要具有防止疾病进展到晚期的潜力的其他治疗剂。对非编码RNA(ncRNA)的作用和临床效用的研究是生物学中的重大进步,其仅最低限度地应用于AMD。在下面的综述中,我们讨论了ncRNA作为生物标志物和治疗靶点在AMD中的临床相关性。
Age-related macular degeneration (AMD) is a progressive neurodegenerative disease that is the world’s leading cause of blindness in the aging population. Although the clinical stages and forms of AMD have been elucidated, more specific prognostic tools are required to determine when patients with early and intermediate AMD will progress into the advanced stages of AMD. Another challenge in the field has been the appropriate development of therapies for intermediate AMD and advanced atrophic AMD. After numerous negative clinical trials, an anti-C5 agent and anti-C3 agent have recently shown promising results in phase 3 clinical trials, in terms of slowing the growth of geographic atrophy, an advanced form of AMD. Interestingly, both drugs appear to be associated with an increased incidence of wet AMD, another advanced form of the disease, and will require frequent intravitreal injections. Certainly, there remains a need for other therapeutic agents with the potential to prevent progression to advanced stages of the disease. Investigation of the role and clinical utility of non-coding RNAs (ncRNAs) is a major advancement in biology that has only been minimally applied to AMD. In the following review, we discuss the clinical relevance of ncRNAs in AMD as both biomarkers and therapeutic targets.
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