Treatment of PD with Nucleic Acid Medicine Using Antisense Oligonucleotides and RNA Interference

Neurodegeneration in PD can be induced by abnormal or excessive levels of α-synuclein protein, suggesting that suppressing the expression of its gene SNCA may prevent further neurodegeneration. a therapeutic approach using ribozyme expressed by adeno-associated virus (AAV) has been attempted to degrade SNCA mRNA and allowed the survival of tyrosine hydroxylase (TH)–positive neurons in the substantia nigra of rats challenged with the dopaminergic toxin 1-methyl-4-phenylpyridinium (MPP⁺). In addition, other methods that use short hairpin RNA (shRNA) and short interfering RNA (siRNA) to suppress SNCA expression have been tested (1).

Reference

1. Nakamori M, Junn E, Mochizuki H, Mouradian M. Nucleic Acid–Based Therapeutics for Parkinson’s Disease. Neurotherapeutics. 2019;16(2):287-298.

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