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N-of-1
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antisense oligonucleotide
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exon skipping
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protocol
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Aartsma-Rus, A.
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Yu, T.W.
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Roon-Mom, W. van
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Consensus guidelines for the design and in vitro preclinical efficacy testing N-of-1 exon skipping antisense oligonucleotides
Consensus guidelines for the design and in vitro preclinical efficacy testing N-of-1 exon skipping antisense oligonucleotides
Whole-genome sequencing holds the key to the success of gene-targeted therapies COMMENT