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Partners
Eberhard Karls Universität Tübingen, Germany (UT)
Leiden University Medical Center, The Netherlands (LUMC)
BianoGMP GmbH, Gera, Germany (Biano)
Ludwig-Maximilians-Universität München, Germany (LMU)
BioTalentum Ltd., Gödöllő, Hungary (BIOT)
mHealth Technologies, Bologna, Italy (MHT)
Uppsala Universitet, Uppsala, Sweden (UU)
Universitätsklinikum Heidelberg, Germany (UKHD)
University of Cambridge, United Kingdom (UCAM)
N=1 Collaborative, inc.
FITT gGmbH, Saarbrücken, Germany
AstraZeneca AB, Göteborg, Sweden
EURORDIS – Rare Diseases Europe
NMI Technologietransfer GmbH, Reutlingen, Germany
Charles River Laboratoiries, Den Bosch B.V., The Netherlands
CeGaT GmbH, Tübingen, Germany
Grasshopper Films GbR, Tübingen, Germany
Technical University Ilmenau, Germany
Alma Mater Studiorum Universita Di Bologna, Italy
MATE Hungarian University of Agriculture and Life Sciences, Hungary
University Medicine Essen, Germany
Agentschap College ter Beoordeling van Geneesmiddelen, Utrecht
Team
Aartsma-Rus, Annemieke (LUMC)
Chiari, Lorenzo (UNIBO)
Coenen, Tineke (LUMC)
Dinnyés, András (BIOT)
Graessner, Holm (UT)
Günther, Rolf (Biano)
Hooker, Andrew (UU)
Horvath, Rita (UCAM)
Karlsson, Mats (UU)
Marckmann, Georg (LMU)
Mellone, Sabato (MHT)
Pöhlmann, Tobias (Biano)
van Roon-Mom, Willeke (LUMC)
Schober, Andreas (TUIL)
Schüle, Rebecca (UKHD)
Synofzik, Matthis (UT)
Tacconi, Carlo (MHT)
Zana, Melinda (BIOT)
PhD Projects
DC1: Pipeline to identify cryptic splice variants and skippable exons at large scale
DC2: Small scale ASO production
DC3: Clinical, research and health care ethics for tailored genetic treatments
DC4: Development and efficacy testing of a cryptic splice ASO (use case ATM)
DC5: Development and efficacy testing of an exon skipping ASO (use case BPAN)
DC6: ASO iPSc toxicity screen in vitro
DC7: ASO safety studies in rat in an academic setting
DC8: Individually tailored digital-motor outcomes in real life
DC9: Individually tailored molecular treatment response profiling
DC10: Statistical methodologies to evaluate efficacy of tailored single-patient treatments
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DC9: Individually tailored molecular treatment response profiling
University of Cambridge
