A novel peptide-based strategy to enhance GBA1 expression for treating Parkinson’s disease

8.5
来源: Nature 关键字: mRNA
发布时间: 2025-11-20 00:08
摘要:

Hirunipin 4, a peptide derived from leeches, has been identified as a novel therapeutic candidate for Parkinson's disease. This study demonstrates that hirunipin 4 enhances GBA1 expression, increases glucocerebrosidase (GCase) activity, and reduces pathological α-synuclein aggregation in neuronal models. Mechanistically, it promotes nuclear translocation of TFEB, a key regulator of lysosomal function, and stabilizes GCase protein levels. The findings suggest that hirunipin 4 could serve as a versatile treatment option for addressing lysosomal dysfunction in neurodegenerative diseases.

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关键证据

Hirunipin 4 significantly enhanced GCase protein levels and enzymatic activity.
The peptide reduced α-synuclein aggregation and improved neuronal viability.
The study provides a novel therapeutic strategy for restoring lysosomal function in Parkinson's disease.

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Hirunipin 4, a peptide derived from leeches, has been identified as a novel therapeutic candidate for Parkinson's disease. This study demonstrates that hirunipin 4 enhances GBA1 expression, increases glucocerebrosidase (GCase) activity, and reduces pathological α-synuclein aggregation in neuronal models. Mechanistically, it promotes nuclear translocation of TFEB, a key regulator of lysosomal function, and stabilizes GCase protein levels. The findings suggest that hirunipin 4 could serve as a versatile treatment option for addressing lysosomal dysfunction in neurodegenerative diseases.

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