Antisense oligonucleotides, which are short sequences of DNA or RNA that can silence or activate mutated genes to rebalance proteins, might prove to be long-awaited therapies for Huntington's, Alzheimer's, Parkinson's and other neurodegenerative diseases. Scientists have been working on ASOs to target genetic diseases since 1978 and have overcome early problems with toxicity, stability and potency, and nusinersen, the first FDA-approved ASO, has been used in more than 10,000 children with spinal muscular atrophy.
ASOs promising for patients with neurological diseases
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