10 PhD fellowships in Gene Therapy of Rare Diseases in EU funded Doctoral Network (# of pos: 10)

Updated: 2 months ago
Job Type: FullTime
Deadline: 20 Oct 2022

Our goal
Rare diseases are affecting more than 30 Mio people in the EU and are in most cases caused by genetic mutations. The only curative treatment for patients suffering from these diseases is gene therapy, but many technical problems still need to be solved before safe and affordable somatic gene therapy becomes a clinical routine. The aim of the EU funded Doctoral Network “Gene Therapy of Rare Diseases” (GetRadi) is to train 10 PhD students to become exceptional experts in gene therapy of rare diseases. By ambitious research projects, the students will aim to overcome major technical challenges that still prevent the widespread use of somatic gene therapy in the clinic. All projects are embedded in a tight and synergistic academic-industrial collaboration with the goal to develop marketable products for the application of gene therapy. This GetRadi training will allow young scientists to spearhead research in gene therapy of rare diseases in academia and industry in their future career.

Our partners
The GetRadi network consists of 9 Principal Investigators at 5 European Universities or Research Institutions and 2 industrial partners, who host the PhD students. All PIs have excellent experience in their respective field of research and a strong interest in genome editing and gene therapy.

Our projects
The 10 GetRadi PhD positions will start March 2023. The Doctoral Network is funded by the Horizon Europe program as part of the Marie Sklodowska-Curie Actions. The general objectives of the GetRadi research projects are to improve the transfer of genome editing tools to the target cells, to optimize CRISPR genome editing or alternatives of it, and to reduce genome editing safety risks. The specific topics of the projects are:

  • Exosome mediated gene therapy of Diamond Blackfan anemia (Prof. Radek Sedlacek, Institute of Molecular Genetics of the Czech Academy of Sciences, Prague, CZ)
  • Adenovector particles mediated gene therapy of Duchenne Muscular Dystrophy (Dr. Manuel Gonc¸alves, Leiden University Medical Center, Leiden, NL)
  • Lipid nanoparticle mediated gene therapy of Hyper IgE syndrome (Dr. Claudio Mussolino, Medical Center University of Freiburg, Freiburg, DE)
  • Gene therapy of Recessive dystrophic epidermolysis bullosa (Prof. Hans Wandall, University of Copenhagen, Copenhagen DK)
  • Gene therapy of Cystic fibrosis (CF) by PRINS prime editing (Dr. Marcello Maresca, AstraZeneca, Gothenburg, SE)
  • Gene therapy of rare neurodevelopmental disorders by a novel Base editing method (Dr. Ralf Ku¨hn, Max Delbrück Centre for Molecular Medicine, Berlin, DE)
  • Identification of epigenetic and F-actin regulators improving the efficiency of ex vivo gene therapy (Prof. Cord Brakebusch, University of Copenhagen, Copenhagen DK)
  • Optimisation of GMP-compliant automated hematopoietic stem cell engineering on the CliniMACS Prodigy (Prof. Eleni Papanikolaou, Miltenyi Biotech, Bergisch Gladbach, DE)
  • Increased safety of CRIPSR-Cas9 dependent gene therapy by novel Cas9 variants with low immunogenicity (Dr. Roberto Nitsch, AstraZeneca, Gothenburg, SE)
  • Easy monitoring of ex vivo and in vivo gene editing efficiency by a novel fluorescent reporter mouse for HDR and NHEJ mediated DNA repair (Prof. Cord Brakebusch, University of Copenhagen, Copenhagen DK)

For more information about the projects and partners, required qualifications of the applicants and application procedure, please select the hyperlinks of the respective project listed above.

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