STSM – year two reports


qPCR quantification of NOTCH3
Maurice Overzier

From June 16th till June 20th I have visited the lab of Dr. Bernard Khoo (University College London, Division of Medicine to work with Dr. Petra Disterer at the Institute of Liver and Digestive Health. The project I am working on focuses on developing …



Follow up mission to investigate miMC as a vector for exon-skipping
Sofia Stenler

In a previous STSM, I travelled to the UK initiate a collaboration with Professor Wells and his lab at the Royal Veterinary College. There we performed experiments in the mdx dystrophic mice model with the main aim to get a proof of principle of the pl …



Ataxin-3 exon skipping in iPSC-derived neurons
Melvin Evers

From the 8th until the 23rd of December 2014, I have visited the Institute of Reconstructive Neurobiology, Life and Brain Center of the University of Bonn in Germany, using funding from the short term scientific mission (STSM) program from COST Action …



Functional testing in mdx mice
Maurice Overzier

University of Bari “Aldo Moro” Department of Pharmacy – Drug sciences Maurice Overzier, research technician at the LUMC in Leiden, the Netherlands From January 24th until January 30th I have visited the Section of Pharmacology in the University of Bari …



Quantification of antisense-mediated exon skipping in DMD by RT-PCR
Iker Garcia

Antisense-mediated exon skipping in Duchenne Muscular Dystrophy aims to restore the expression of dystrophin protein. However, accurate quantification of dystrophin expression is challenging and, therefore, it is difficult to evaluate the outcome of th …



Dystrophin expression in relation to myonuclear organisation
Julien Ochala

From May 11th to May 15th 2015, I have been to the lab of Dr. Annemieke Aartsma-Rus (DMD Genetic Therapy Group, Department of Human Genetics, LUMC, the Netherlands) and worked with Dr. Maaike Van Putten. The project we are working on focuses on dystrop …