Learning the lab techniques associated with exon skipping therapy using antisense oligonucleotides

Jakub Malcher, Germany

1st year PhD student at Free University of Berlin & University of Versailles

In April 2014 I was on my Short Term Scientific Mission (STSM) at the Department of Laboratory Medicine at Karolinska Insitute in Stockholm Sweden where I was hosted by Dr. Samir El-Andaloussi. The goal of my STSM was to learn the lab techniques applied in my projects on the exon-skipping therapy using antisense oligonucleotides tricycloDNA (tcDNA) for the DMD and Dysferlinopathy disease models. Apart from the investigation of the therapeutic potential of tcDNA, I am interested in understanding the uptake mechanism of these oligonucleotides into the mammalian cell. Studying these mechanisms requires special set of skill which I was able to acquire on my STSM. During the two weeks of my stay I learnt how to perform the Nano Tracking Analysis (NTA), In Vivo Imagaing (IVIs) and the basics of HPLC. The trainings involved both practical aspects and data analysis. I was also trained how to setup in vitro experiments using endocytosis inhibitors. This STSM not only helped me to improve my scientific skills but also enabled further information dissemination in the lab, where I am currently doing my PhD. Furthermore, I had a chance to participate in the COST Workshop on “Antisense Chemistry and Toxicology”, which was taking place at the same time in Stockholm. This was a great opportunity to meet the members of the Exon Skipping COST Action and to hear the latest developments in this field. Hence, the STMS in Karolinska Institute gave me a good practical and theoretical background for my further research and contributed a lot to the development of my scientific network in the field that I am currently working in.

April 2013