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New components for antisense gene therapy show promise in treating spinal muscular atrophy
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New components for antisense gene therapy show promise in treating spinal muscular atrophy

by  Skolkovo Institute of Science and Technology Credit: Pixabay/CC0 Public Domain Skoltech researchers and their colleagues from Russia and the UK investigated the safety and efficacy of new chemistry in antisense oligonucleotides used to treat spinal muscular atrophy (SMA), a debilitating genetic disease. Their results may lead to the development of drugs with less toxicity and fewer injections...