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Nusinersen is an ASO used for the treatment of spinal muscular atrophy (SMA). Using your knowledge of antisense oligonucleotide development, identify the key aspects of
Nusinersen is an ASO used for the treatment of spinal muscular atrophy (SMA). Using your knowledge of antisense oligonucleotide development, identify the key aspects of the design of this oligonucleotide. Suggest possible chemical modifications that can be introduced to improve its targeting properties in cellular environments. Use schematics and provide reasoning for all changes made
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