How can gene therapy be used to treat PLA2G6 Associated Neurodegeneration?
DOI:
https://doi.org/10.58445/rars.1699Keywords:
PLA2G6, CRISPR-Cas9, Parksinson’s, neurodegeneration, gene therapyAbstract
Neurodegenerative diseases rank among the most prevalent and challenging health burdens globally, characterized by progressive neuronal degeneration. Mutations in the PLA2G6 gene, encoding the iPLA2β enzyme critical for cellular phospholipid metabolism underlie severe neurodegenerative syndromes such as infantile neuroaxonal dystrophy (INAD) and dystonia-parkinsonism. Advances in gene therapy, notably CRISPR-Cas9 technology, offer a promising approach to target and correct underlying genetic mutations. CRISPR-Cas9 enables precise modification of the mutated PLA2G6 gene, potentially restoring normal enzyme function and halting disease progression. However, effective delivery of CRISPR components to the central nervous system remains a significant challenge, necessitating innovative strategies such as intracranial viral vector delivery for targeted gene editing. This paper explores the molecular mechanisms of PLA2G6-related neurodegenerative diseases, discusses the application of CRISPR-Cas9 for genetic correction, and evaluates intracranial viral vector delivery as a feasible delivery method. By exploring these approaches, this research aims to contribute to the development of novel therapies aimed at addressing the genetic causes of neurodegenerative diseases associated with PLA2G6 mutations, potentially offering new hope for patients affected by these devastating conditions.
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