Date: Sept. 11, 2023 

Source: Guangdong-HongKong-Macao Institute of CNS Regeneration 

Author: Yan Sen

Translator: Wu Xueyi

JNU Profs. Li Shihua and Li Xiaojiang, researcher Yan Sen and their team have reported a new therapeutic strategy to treat Huntington's Disease. The internationally renowned journal Advanced Science (IF=15.1) published their research results, titled A Specific Mini-Intrabody Mediates Lysosome Degradation of Mutant Huntingtin, online on Sept. 9 (https://onlinelibrary.wiley.com/doi/full/10.1002/ advs. 202301120).

Huntington's disease is a single-gene mutated autosomal-dominant neurodegenerative disease caused by the intracellular accumulation of mutant huntingtin (mHTT) that aggregates in the central nervous system and causes neuronal damage and death. Accumulation of misfolded proteins leads to many neurodegenerative diseases; therefore, the most direct and effective way to treat these diseases is to lower or remove mutant proteins.

(Screenshot of the paper)

The corresponding authors are Profs. Li and Li and Yan Sen of JNU's Guangdong-Hong Kong-Macao Institute of CNS Regeneration. The first authors are doctoral students Li Caijuan and Lin Yingqi. The research has been supported by the National Key Research and Development Program of China, the National Natural Science Foundation of China, the Guangzhou Key Research Program on Brain Science, Jinan University, the Guangdong Department of Science and Technology, the Guangzhou Municipal Science and Technology Bureau, and the Guangdong Key Laboratory of Non-Human Primate Research.