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基于RNA的治疗神经发育障碍:用于分子校正的创新工具

Denise Drongitis¹, Lucia Verrillo¹, Alberto de Bellis^2,3

¹Institute of Genetics and Biophysics "Adriano Buzzati-Traverso", CNR, Naples, Italy.

Frontiers in molecular biosciences

|November 17, 2025

概括

基于RNA的疗法为神经发育障碍 (NDD) 提供有针对性的治疗方法,如智力障碍 (ID) 和自闭症谱系障碍 (ASD). 这些创新的RNA分子,包括反感性寡核化物 (ASOs),在纠正基因突变和恢复神经元功能方面表现有前途.

科学领域:

神经科学是一个神经科学.
遗传学是一个遗传学.
分子生物学分子生物学

背景情况:

神经发育障碍 (NDD) 通常源于特定的遗传突变.
目前许多NDD的治疗选择有限,特别是在综合征形式.
恢复正常的RNA和蛋白质表达是一种关键的治疗策略.

研究的目的:

审查基于RNA的NDD治疗方法的进展.
突出各种RNA分子在治疗遗传疾病方面的潜力.
探索RNA医学在智力障碍,自闭症谱系障碍和发育性脑病的应用.

主要方法:

综述各种基于RNA的治疗类别:反感性寡核酸 (ASOs),抗NAT,SINEUPs,RNAi,ExSpeU1s和saRNA.
对针对NDDs特定基因突变的治疗应用的分析.
在临床前和临床研究中检查RNA分子的疗效.

主要成果:

基于RNA的疗法对综合征性ID (脆弱X,MECP2重复,FOXG1,安吉尔曼综合征) 有希望.
目前正在探索与ASD相关的疾病 (例如CHD8突变) 的研究RNA方法.
正在开发RNA分子,通过向CDKL5,SCN1A等基因来解决DEE中严重的耐药性.

结论:

RNA技术为NDD提供了针对基因的特定和灵活的干预措施.
RNA医学领域正在迅速扩大,为分子校正提供了新的希望.
本综述是研究人员和临床医生关于NDDs的创新RNA工具的资源.

关键词:

RNA代谢RNA代谢的过程动物模型动物模型调节基因表达调节基因表达遗传性的NDDs是没有发生的.简单地说,这是一个简单的缺陷.翻译翻译翻译翻译翻译翻译

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