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Research Review

Showing 1–1 of 1 articles · Keyword: gene replacement Clear keyword

Frontiers in molecular neuroscience2026ReviewNon-viral Gene Delivery

1. Restoring Parkin function: an AAV gene therapy approach for early-onset Parkinson's disease

Basu S, Demarest Tg, Gattone Nj, Gilsrud Aj, Wicks B, Khatiwada A, Et Al.

Biallelic loss-of-function mutations in PRKN gene (encoding Parkin protein) cause early-onset Parkinson's disease (EOPD). Parkin is a crucial component of the PINK1-Parkin pathway, which marks damaged mitochondria for degradation via mitophagy. Without functional Parkin, damaged mitochondria accumulate, causing…

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