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Synthesis, Biological Activity, and Molecular Dynamics Simulations of LNA-Charge Neutral Linkages for Enhanced Splice-Switching Antisense Oligonucleotides

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Antisense oligonucleotides are promising therapeutic agents for a range of diseases, having found special clinical success for splice-switching genetic conditions such as spinal muscular atrophy and Duchenne muscular dystrophy. However, novel chemistries are s... ...