ArticleNPJ Parkinson's disease2025
A novel peptide-based strategy to enhance GBA1 expression for treating Parkinson's disease.
Article in NPJ Parkinson's disease, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Who cites it
1 citing paper in PubMed.
- Human pluripotent stem cell engineering with CRISPR-Cas9 for Parkinson's disease.Experimental & molecular medicine · 2026Review
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Authors and funding
15 authors.
Funding
Abstract
Parkinson's disease (PD) is a progressive neurodegenerative disorder characterized by α-synuclein aggregation and lysosomal dysfunction, with GBA1 mutations representing the most common genetic risk factor. Reduced glucocerebrosidase (GCase) activity is observed in both familial and sporadic PD, promoting α-synuclein accumulation and neuronal toxicity. Here, we developed a GBA1-HiBiT tagged knock-in HEK293T reporter system, providing a highly sensitive, real-time quantitative measurement of GCase dynamics. From a leech-derived peptide library, we identified a cell-penetrating peptide hirunipin 4 that significantly enhanced GCase protein levels and enzymatic activity. Moreover, hirunipin 4 alleviated α-synuclein preformed fibrils (PFF)-induced lysosomal dysfunction and glucosylceramide accumulation. Thus, hirunipin 4 was able to reduce PFF-induced pathological α-synuclein accumulation and neurotoxicity in both SH-SY5Y and primary cortical neurons. Mechanistically, hirunipin 4 promoted nuclear translocation of TFEB and enhanced GCase protein stability. These findings highlight the utility of the GBA1-HiBiT platform for peptide-based screening and identify hirunipin 4 as a promising candidate for restoring lysosomal function in PD.
Identifiers
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.