Molecular therapy for polyQ disorders: from bench to clinical trials

João de Sousa-Lourenço,Ana C. Silva,Luís Pereira de Almeida,Rui J. Nobre

Trends in Molecular Medicine（2024）

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摘要

Polyglutamine (polyQ) disorders are monogenic neurodegenerative disorders. Currently, no therapies are available for this complex group of disorders. Here, we aim to provide an overview of recent promising preclinical studies and the ongoing clinical trials focusing on molecular therapies for polyQ disorders.

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关键词

polyQ disorders,molecular therapy,clinical trials,Huntington’s disease,spinocerebellar ataxia type 1,spinocerebellar ataxia type 3

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