Recent advances in RNA-based therapeutics for neurodevelopmental disorders

IF 3.7 2区生物学 Q2 CELL BIOLOGY

Current Opinion in Genetics & Development Pub Date : 2025-03-21 DOI:10.1016/j.gde.2025.102339

Harini P Tirumala , Huda Y Zoghbi

引用次数: 0

Abstract

A significant proportion of neurodevelopmental disorders (NDDs) are caused by gain-of-function (GOF) or loss-of-function (LOF) of specific genes. Strategies to normalize disease gene expression offer therapeutic potential for these disorders. The success and approval of RNA-based therapeutics for various disorders have led to a surge in RNA-based therapeutic research for NDDs with antisense oligonucleotides leading the field. This review discusses recent advances in therapeutic strategies that target pre-mRNA or mRNA for GOF and LOF NDDs that have promising preclinical evidence. These developments highlight important considerations and exciting future avenues for the development of therapies for NDDs.

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基于rna的神经发育障碍治疗的最新进展

很大一部分神经发育障碍（ndd）是由特定基因的功能获得（GOF）或功能丧失（LOF）引起的。使疾病基因表达正常化的策略为这些疾病提供了治疗潜力。基于rna的各种疾病治疗方法的成功和批准，导致了以反义寡核苷酸为主导的基于rna的ndd治疗研究的激增。本文综述了以pre-mRNA或mRNA为靶点的GOF和LOF ndd治疗策略的最新进展，这些策略具有良好的临床前证据。这些发展突出了ndd治疗发展的重要考虑和令人兴奋的未来途径。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Current Opinion in Genetics & Development 生物-细胞生物学

CiteScore

7.90

自引率

0.00%

发文量

102

审稿时长

1 months

期刊介绍： Current Opinion in Genetics and Development aims to stimulate scientifically grounded, interdisciplinary, multi-scale debate and exchange of ideas. It contains polished, concise and timely reviews and opinions, with particular emphasis on those articles published in the past two years. In addition to describing recent trends, the authors are encouraged to give their subjective opinion of the topics discussed. In Current Opinion in Genetics and Development we help the reader by providing in a systematic manner: 1. The views of experts on current advances in their field in a clear and readable form. 2. Evaluations of the most interesting papers, annotated by experts, from the great wealth of original publications.[...] The subject of Genetics and Development is divided into six themed sections, each of which is reviewed once a year: • Cancer Genomics • Genome Architecture and Expression • Molecular and genetic basis of disease • Developmental mechanisms, patterning and evolution • Cell reprogramming, regeneration and repair • Genetics of Human Origin / Evolutionary genetics (alternate years)