Objective
More than 300 million people worldwide are suffering from more than 6000 rare diseases. Nearly all of these rare diseases are caused by a single inherited mutation and cannot be treated effectively. Repair of the defective gene by gene editing is the only possible curative therapy. However, only for very few rare diseases such gene editing therapy has reached the clinic. The “Gene therapy of Rare Diseases” (GetRadi) consortium aims now to contribute strongly to the establishment of more gene therapies for rare diseases. This will be accomplished by training of future leaders in gene therapy of rare diseases preforming ambitious research projects, with the following objectives: (i) Improving transfer of genome editing tools to target cells, (ii) improving gene editing efficiency, and (iii) improving safety of gene therapy. Strong participation of the pharmaceutical industry to research projects and training, development of novel in vitro and in vivo models for rare diseases to test gene therapies in relevant settings, and application of several unique genome editing tools developed by the applicants to the treatment of rare diseases are hallmarks of the network. GetRadi brings together strong industrial beneficiaries (AstraZeneca, Miltenyi Biotec) supervising 3 ESR, highly innovative academic beneficiaries supervising 7 ESR, 3 additional industrial partners, and 4 student enrolling universities. Professional outreach training with access to widely used social media channels will be provided by the partner “European Consortium for Communicating Gene and Cell Therapy Information”. Complementary unique expertise that is spread efficiently by network-wide training and secondments, excellent quality of supervision and intersectoral interactions, innovative transferable skill training, and efficient consortium management will result in an exceptional training of future leaders in gene therapy of rare diseases.
Fields of science
Programme(s)
- HORIZON.1.2 - Marie Skłodowska-Curie Actions (MSCA) Main Programme
Funding Scheme
HORIZON-TMA-MSCA-DN - HORIZON TMA MSCA Doctoral NetworksCoordinator
1165 Kobenhavn
Denmark
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Participants (6)
151 85 Sodertaelje
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79106 Freiburg
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2333 ZA Leiden
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13125 Berlin
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142 20 Praha 4
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51429 Bergisch Gladbach
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Partners (7)
Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
2333 CH Leiden
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The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.
Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
22363 Lund
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The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.
Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
EH8 9YL Edinburgh
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Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
10117 Berlin
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Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
116 36 Praha 1
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Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
79098 Freiburg
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Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.
80138 Napoli
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