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ASOs are short, synthetic strands of nucleotides that can bind to mRNA and modulate protein expression. Tofersen , an ASO targeting SOD1 mRNA, was recently approved for SOD1-ALS. Clinical trials have shown that reducing SOD1 protein levels can slow disease progression. Similarly, trials are underway for ASOs targeting C9orf72 and ATXN2 . amoytoge new
If you're looking for information on a specific topic, could you please provide more keywords or details? That way, I can try to assist you in finding a relevant paper or information. Tofersen , an ASO targeting SOD1 mRNA, was
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