Yahya E. Jad

Timing matters: exon skipping therapy is most effective when initiated early in a mouse model of Duchenne muscular dystrophy

This study shows that exon skipping therapy is more effective when started early in adult mdx mice than in aged mice, leading to better dystrophin restoration and transcriptomic changes.

Toward a model of uORF-mediated translational control: An integrated bioinformatic and experimental approach

The paper combines experimental and computational methods to model how uORFs regulate translation, identifying key features and interactions that influence their repressive activity.

Dysregulated MEG3 in Myotonic Dystrophy 1: nuclear retention, pathological role, and therapeutic correction by antisense conjugates.

This study shows MEG3 is upregulated in DM1 and exhibits nuclear retention, with antisense conjugates correcting MEG3 levels in mice.

uORF-targeting steric block antisense oligonucleotides do not reproducibly activate RNASEH1 expression

The paper tests uORF-targeting ASOs for RNASEH1 upregulation but finds no reproducible activation, instead observing downregulation in some cases.