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Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
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Splicing modulation therapy in the treatment of genetic diseases

Virginia Arechavala-Gomeza1, Bernard Khoo2, Annemieke Aartsma-Rus3

  • 1Neuromuscular Disorders Group, BioCruces Health Research Institute, Barakaldo, Bizkaia, Spain.

The Application of Clinical Genetics
|December 16, 2014
PubMed

Keywords:
alternative splicingantisense oligonucleotidescryptic splicingsplicingtherapy

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