Pharmacology & ToxicologybioRxivPreprint
Anti-Sense Oligonucleotide as a Therapeutic for Synucleinopathies: Pharmacokinetic, Safety and Efficacy Evaluation
Rijwan Uddin Ahammad, Brian J. Spencer, Bao Quach…·Robert A. Rissman
This study evaluates an antisense oligonucleotide (ASO) therapy for synucleinopathies, showing effective CNS delivery and reduction of α-synuclein levels in a mouse model.
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