COST CA 17103
Delivery of Antisense RNA Therapeutics
Antisense oligonucleotides (ASOs) are a new class of drugs that, through very specific targeting, could correct genetic defects for rare inherited diseases, modulate autoimmune or neurodegenerative diseases or target tumors or viruses. However, only a few of such drugs are currently in the market and they have been less effective as expected. The main hurdle for their efficacy seems to be their deficient delivery to target tissues but, while translational research on ASO is surging, very little is known about the mechanisms by which ASOs are taken up by different tissues and specific cells.
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