Purpose of the STSM:
The aim of this STSM was to validate exon skipping efficiency using an antisense oligonucleotide (AON) to skip exon 51 (the same we have tried before in DMD human myoblasts) and to test dystrophin expression comparing control and DMD pig cultures before and after being treated with the AON.
Grantee name: Patricia Soblechero Martin (BioCruces Health Research Institute, Barakaldo, Bizkaia, Spain).
STSM start and end date: 2019-03-03 to 2019-03-30
Title: Validation of a pig cell culture to evaluate antisense therapies for DMD
Host lab: Sabine Krause. Friedrich Baur Institute, Munich, Germany.