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Potential of cell-penetrating peptide-conjugated antisense oligonucleotides for the treatment of SMA复制

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J Leckie, T Yokota
Molecules, 2024
mdpi.com
Spinal muscular atrophy (SMA) is a severe neuromuscular disorder that is caused by
mutations in the survival motor neuron 1 (SMN1) gene, hindering the production of functional …

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