Powered by OpenAIRE graph
Found an issue? Give us feedback

GetRadi

Gene Therapy of Rare Diseases
Funder: European CommissionProject code: 101072427 Call for proposal: HORIZON-MSCA-2021-DN-01
Funded under: HE | HORIZON-TMA-MSCA-DN Funder Contribution: 2,785,810 EUR
Description

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.

Data Management Plans
Powered by OpenAIRE graph
Found an issue? Give us feedback

Do the share buttons not appear? Please make sure, any blocking addon is disabled, and then reload the page.

All Research products
arrow_drop_down
<script type="text/javascript">
<!--
document.write('<div id="oa_widget"></div>');
document.write('<script type="text/javascript" src="https://www.openaire.eu/index.php?option=com_openaire&view=widget&format=raw&projectId=corda_____he::ee090197e731b2ff0f8aa4eca3044e8e&type=result"></script>');
-->
</script>
For further information contact us at helpdesk@openaire.eu

No option selected
arrow_drop_down