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However, to date, oligonucleotide therapeutics are still facing some technological obstacles, such as how to achieve efficient oligonucleotide delivery to target organs or tissues, how to overcome off-target interactions [4952], and how to address the sequence- and chemistry-dependent toxicity and saturation of endogenous RNA processing pathways [53, 54]

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This means they can penetrate the blood-brain (or blood-SCF) barrier and reach neurons directly, enabling the drug to exhibit organ-specific combined effects in the brain