RNA therapeutics for neurological diseases.

IF 6.7 2区医学 Q1 Medicine

British medical bulletin Pub Date : 2023-09-12 DOI:10.1093/bmb/ldad010

Ilaria Brentari, Mariia Zadorozhna, Michela Alessandra Denti, Elisa Giorgio

{"title":"RNA therapeutics for neurological diseases.","authors":"Ilaria Brentari, Mariia Zadorozhna, Michela Alessandra Denti, Elisa Giorgio","doi":"10.1093/bmb/ldad010","DOIUrl":null,"url":null,"abstract":"Introduction: Ribonucleic acid (RNA) therapeutics are a new class of drugs whose importance is highlighted by the growing number of molecules in the clinic.Sources of data: We focus on RNA therapeutics for neurogenetic disorders, which are broadly defined as diseases with a genetic background and with at least one clinical sign affecting the nervous system. A systematic search identified 14 RNA drugs approved by FDA and many others in development.Areas of agreement: The field of RNA therapeutics is changing the therapeutic scenario across many disorders.Areas of controversy: Despite its recent successes, RNA therapeutics encountered several hurdles and some clinical failures. Delivery to the brain represents the biggest challenge.Growing points: The many advantages of RNA drugs make the development of these technologies a worthwhile investment.Areas timely for developing research: Clinical failures stress the importance of implementing clinical trial design and optimizing RNA molecules to hold the promise of revolutionizing the treatment of human diseases.","PeriodicalId":9280,"journal":{"name":"British medical bulletin","volume":"147 1","pages":"50-61"},"PeriodicalIF":6.7000,"publicationDate":"2023-09-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"1","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"British medical bulletin","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1093/bmb/ldad010","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"Medicine","Score":null,"Total":0}

引用次数: 1

Abstract

Introduction: Ribonucleic acid (RNA) therapeutics are a new class of drugs whose importance is highlighted by the growing number of molecules in the clinic.

Sources of data: We focus on RNA therapeutics for neurogenetic disorders, which are broadly defined as diseases with a genetic background and with at least one clinical sign affecting the nervous system. A systematic search identified 14 RNA drugs approved by FDA and many others in development.

Areas of agreement: The field of RNA therapeutics is changing the therapeutic scenario across many disorders.

Areas of controversy: Despite its recent successes, RNA therapeutics encountered several hurdles and some clinical failures. Delivery to the brain represents the biggest challenge.

Growing points: The many advantages of RNA drugs make the development of these technologies a worthwhile investment.

Areas timely for developing research: Clinical failures stress the importance of implementing clinical trial design and optimizing RNA molecules to hold the promise of revolutionizing the treatment of human diseases.

查看原文本刊更多论文

神经系统疾病的RNA疗法。

引言：核糖核酸（RNA）治疗是一类新型药物，其重要性因临床上分子数量的增加而凸显。数据来源：我们专注于神经源性疾病的RNA治疗，神经源性障碍被广泛定义为具有遗传背景且至少有一种临床症状影响神经系统的疾病。一项系统搜索确定了14种已获美国食品药品监督管理局批准的RNA药物和许多其他正在开发中的药物。一致的领域：RNA治疗领域正在改变许多疾病的治疗方案。争议领域：尽管RNA疗法最近取得了成功，但它遇到了一些障碍和一些临床失败。输送到大脑是最大的挑战。成长点：RNA药物的许多优势使这些技术的开发成为一项值得投资的投资。及时开展研究的领域：临床失败强调了实施临床试验设计和优化RNA分子的重要性，以实现人类疾病治疗的革命性。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

求助全文

约1分钟内获得全文求助全文

来源期刊

British medical bulletin 医学-医学：内科

CiteScore

13.10

自引率

1.50%

发文量

审稿时长

>12 weeks

期刊介绍： British Medical Bulletin is a multidisciplinary publication, which comprises high quality reviews aimed at generalist physicians, junior doctors, and medical students in both developed and developing countries. Its key aims are to provide interpretations of growing points in medicine by trusted experts in the field, and to assist practitioners in incorporating not just evidence but new conceptual ways of thinking into their practice.