STSM – year four reports

A new method to identify protein interactions with the DMD transcript
Miriam Geissler

During the last months, I had the opportunity to join the research group of Prof. Annemieke Aartsma-Rus from the Leiden University Medical Centre (LUMC) where I could complete my master’s thesis under the supervision of Prof. Janbernd Kirschner from th …

Exon skipping therapy using antisense oligonucleotides
Gisela Gaina

Short term scientific mission (STSM) – From generation of primary muscle culture to exon skipping therapy using antisense oligonucleotides. Gisela Gaina, Romania Department of Biochemistry and Molecular Biology, University of Bucharest, Romania My Shor …

Development of antisense U1 snRNA-mediated therapeutic strategies to modulate splicing in lysosomal storage disorders
Liliana Matos

Lysosomal storage disorders (LSDs) are a group of inherited metabolic disorders that can result in severe and progressive pathology due to a specific lysosomal dysfunction. In several patients, splicing mutations have been identified and some of these …