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Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides
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Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides

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Therapeutic modulation of DMD splicing by blocking exonic splicing enhancer sites with antisense oligonucleotides

A Aartsma-Rus1, A A M Janson, J A Heemskerk

  • 1Department of Human Genetics, DMD Genetic Therapy Group, Leiden University Medical Center, 2333 AL Leiden, The Netherlands.

Annals of the New York Academy of Sciences
|December 6, 2006
PubMed

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