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Cartilaginous tissue regeneration by non-viral gene therapy; taking the hurdles towards efficient delivery

Project description

Non-viral gene therapy for intervertebral disc degeneration

Intervertebral disc (IVD) degeneration is a main cause of chronic low back pain and osteoarthritis of the joints, affecting many individuals worldwide. To restore tissue health in affected patients, the EU-funded CARTHAGO project is working on a non-viral gene therapy approach based on the delivery of oligonucleotides and encoding nucleic acids. This strategy combined with ultrasound overcomes accessibility issues of cartilaginous tissues encountered by traditional drugs and enhances tissue penetration and transfection. The work will be carried out by early-stage researchers who will also acquire transferable skills for a career in the field.

Objective

Chronic low back pain due to intervertebral disc (IVD) degeneration and osteoarthritis (OA) worldwide impact human health and well-being due to pain and impaired mobility. Non-viral gene therapy has great promise as safe and precision treatment to restore IVD and joint tissue health. However, the lack of accessibility of the affected cartilaginous tissues to drugs has inhibited progress in this field. We will finally fulfil the promise of non-viral gene therapy in these diseases. We will do this through educating 15 young researchers in 10 different countries in physics, quality by design (QbD), nucleic acid chemistry, nanomedicine, cartilage and IVD biology, ethics, entrepreneurship and academic transferable skills.
This highly multidisciplinary team will exploit the potential of gene therapy in IVD and joint disease by taking a multi-faceted approach towards the delivery and activity of oligonucleotides and encoding nucleic acids (NA). Cell and tissue delivery will be achieved by nucleic acid modification, innovative nanocarriers and tissue targeting, supported by QbD. Ultrasound will for the first time be used to enhance NA tissue penetration and transfection, and to trigger local release from systemically administered endogenous albumin-NA conjugates. Imaging tools will be developed to monitor in vivo regeneration, saving numerous animal lives in the future. Moreover, we will set up an educational framework for embedding biomedical ethics in biomedical research and valorisation, providing a firm and sustainable basis for Responsible Research and Innovation for future biomedical science and technology. Participation of a patient organisation and a scientific organisation experienced in the dialogue with society, will ensure researchers at ease in communicating with different audiences. Hence CARTHAGO will deliver the agile scientists freely moving between sectors and firmly anchored in society, required to take science and innovation to the next level in Europe.

Coordinator

UNIVERSITAIR MEDISCH CENTRUM UTRECHT
Net EU contribution
€ 265 619,88
Address
HEIDELBERGLAAN 100
3584 CX Utrecht
Netherlands

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Region
West-Nederland Utrecht Utrecht
Activity type
Higher or Secondary Education Establishments
Links
Total cost
€ 265 619,88

Participants (11)