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Recent advances in antisense oligonucleotide therapy in genetic neuromuscular diseases

1 Jan 2018

Abstract excerpt

Genetic neuromuscular diseases are caused by defective expression of nuclear or mitochondrial genes. Mutant genes may reduce expression of wild-type proteins, and strategies to activate expression of the wild-type proteins might provide therapeutic benefits. Also, a toxic mutant protein may cause cell death, and strategies that reduce mutant gene expression may provide therapeutic benefit. Synthetic antisense...

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PMID: 29720791
DOI: 10.4103/aian.aian_298_17

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