Purpose of the STSM: Duchenne muscular dystrophy is a chronic devastating disorders affecting 1:5000 new born males. It is caused by a lack of dystrophin protein, which is normally expressed in muscle fibers and prevents them from damage during contractions. One of the most promising therapeutic strategies is restoring the disrupted open reading frame of the dystrophin gene through antisense oligonucleotide-(AON) mediated exon skipping. The efficacy of this therapeutic approach significantly depends on optimal delivery; all muscles need to be targeted. In my research, I aim to improve AON delivery through either conjugation of peptides to AONs or the use of modified chemistries. In addition to pharmacokinetic and dynamic studies, I assess muscle performance to determine treatment efficacy.
Grantee name: Maaike van Putten (…)
STSM start and end date: 2020-02-24 to 2020-02-28
Title: Ex vivo force measurement of the diaphragm muscle to assess dystrophin restoration efficacy
Host lab: …