A Splice Intervention Therapy for Autosomal Recessive Juvenile Parkinson’s Disease Arising from Parkin Mutations

A Splice Intervention Therapy for Autosomal Recessive Juvenile Parkinson’s Disease Arising from Parkin Mutations

Parkin-type autosomal recessive juvenile-onset Parkinson’s disease is caused by mutations in the <i>PRKN</i> gene and accounts for 50% of all autosomal recessive Parkinsonism cases. Parkin is a neuroprotective protein that has dual functions as an E3 ligase in the ubiquitin–proteasome sy...

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Bibliographic Details
Main Authors: Dunhui Li, May T. Aung-Htut, Kristin A. Ham, Sue Fletcher, Steve D. Wilton
Format: Article
Language:English
Published: MDPI AG 2020-10-01
Series:International Journal of Molecular Sciences
Subjects:
antisense oligonucleotides
exon skipping
juvenile-onset Parkinson’s disease
precision medicine
Online Access:https://www.mdpi.com/1422-0067/21/19/7282

Internet

https://www.mdpi.com/1422-0067/21/19/7282

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