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Antisense oligonucleotide (ASO) therapy has the potential to treat a variety of neurodegenerative diseases by inhibiting the production of pathogenic proteins or non-coding RNA at the gene level
A few days ago, researchers from Tokyo Medical and Dental University (TMDU), Ionis Pharmaceuticals, and Takeda Corporation have discovered a new method to deliver ASO subcutaneously or intravenously across the blood-brain barrier to the central nervous system
Generally, only hydrophobic molecules with a molecular weight of less than 450 Da can cross the blood-brain barrier
Experimental results show that this cholesterol-coupled HDOs can be widely distributed in the brain, spine and peripheral tissues, the most in the CNS
▲HDOs (BBB-HDOs) that can cross the blood-brain barrier significantly inhibit the expression of target genes in the cerebral cortex (Image source: Credit: Department of Neurology and Neurological Science, TMDU)
Moreover, the research team further proved that the ability of this HDOs to cross the blood-brain barrier did not cause a decrease in the integrity of the blood-brain barrier itself
Reference materials:
[1] From blood to brain: Delivering nucleic acid therapy to the central nervous system.
[2] Nagata et al.
(The original text has been deleted)
