针对遗传疾病的多肽疗法。

IF 6.5 2区医学 Q1 PHARMACOLOGY & PHARMACY

Drug Discovery Today Pub Date : 2024-10-16 DOI:10.1016/j.drudis.2024.104209

Shweta Subramanian, Meenakshi Jain, Rajkumar Misra, Rahul Jain

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引用次数: 0

摘要

遗传性疾病（GDs）的治疗具有挑战性，因为缺乏最佳的治疗方案，而且其潜在的生物过程错综复杂，往往难以理解。现有的治疗方法有限，大多只能缓解症状。迄今为止，用于治疗 GD 的肽类药物数量有限，有几种候选药物正在临床研究中。本综述从机理上深入探讨了 GDs 和肽类药物的潜在目标领域。此外，它还强调了多肽作为基因递送载体、突变检测生物标记物和多肽疫苗在治疗 GDs 方面的作用。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

Peptide-based therapeutics targeting genetic disorders

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Peptide-based therapeutics targeting genetic disorders

Genetic disorders (GDs) are challenging to treat owing to a lack of optimal treatment regimens and intricate and often difficult-to-understand underlying biological processes. Limited therapeutic approaches, which mostly provide symptomatic relief, are available. To date, a limited number of peptide-based drugs for the treatment of GDs are available, and several candidates are under clinical study. This review provides mechanistic insights into GDs and potential target areas where peptide-based drugs are beneficial. In addition, it emphasizes the usefulness of peptides as carriers for gene delivery, biomarkers for mutation detection and peptide-based vaccines for treating GDs.

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来源期刊

Drug Discovery Today 医学-药学

CiteScore

14.80

自引率

2.70%

发文量

293

审稿时长

6 months

期刊介绍： Drug Discovery Today delivers informed and highly current reviews for the discovery community. The magazine addresses not only the rapid scientific developments in drug discovery associated technologies but also the management, commercial and regulatory issues that increasingly play a part in how R&D is planned, structured and executed. Features include comment by international experts, news and analysis of important developments, reviews of key scientific and strategic issues, overviews of recent progress in specific therapeutic areas and conference reports.