The preclinical work on exon skipping for rare diseases is fragmented. Even though many of us work on different diseases, the challenges we face are the same. On the other hand, some of us work on exon skipping for the same disease and we should avoid doing the same work simultaneously.
To harmonize our efforts, and jointly tackle common challenges, regular workshops are organized where participants confidentially discuss hurdles and their latest results.
All our members are involved in this effort.
Planned activities
Our Action has ended in April 2017. As such we have no further planned activities.
Most Recent Activities
Final Action Meeting March 2017 Several back to back meetings were organized by the Action March 20-22 in Porto, Portugal, where we were hosted by local organizers Sandra Alves and Liliana Matos.
- AON design workshop (March 20) Selected researchers participated in an interactive workshop on AON design, organized by Annemieke Aartsma-Rus and Petra Disterer. Participants were taught the guidelines for exon skipping AON design and then had to apply their knowledge to design their own AONs.
- Discussion session on career advice (March 20)Participants had an open discussion on career paths, uncertainties, doubts, risks and opportunities in academia.
- Biomarker workshop (March 20-21) Participants presented on their most recent developments in biomarker work in a confidential setting. Presentations were primarily given by early stage researchers. The meeting was organized and Chaired by Pietro Spitali and Valentina Sardone.
- Q & A session with Art Krieg (March 21) An open forum discussion chaired by Annemieke Aartsma-Rus with keynote lecturer Arg Krieg was held. Discussion topics varied from AON therapy development and inflammatory effects of AONs to a career in industry vs a career in academia.
- Final Action Meeting (March 22) Participants presented the results of the Action, the different working groups and its impact. Art Krieg gave a keynote lecture. Selected early stage researchers presented their STSMs.
 1 – Interaction at the AON design workshop |
 3 – Setting up for the biomarker workshop |
 4 – Art Krieg |
 5 – Maria Ines Alvelos presenting her STSM |
Activities so far
June 2016 – ExPRESS summer school 2016
February 2016 – Workshop on delivery and delivery to the CNS
October 2015 – Exon skipping session at the OTS meeting
June 2015 – ExPRESS summer school
May 2015 – Animal models meeting
April 2015 – Stakeholder workshop at EMA
April 2015 – Presenting scientific work to patients and parents
April 2014 – Chemistry and Toxicology meeting
