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

Descripción del proyecto

Genoterapia no vírica para tratar la discopatía degenerativa

La discopatía degenerativa es una de las principales causas de la lumbalgia crónica y la artrosis articular, que afecta a muchas personas en todo el mundo. Para restaurar la salud del tejido en los pacientes afectados, el proyecto CARTHAGO, financiado con fondos europeos, está trabajando en un enfoque de genoterapia no vírica basado en la administración de oligonucleótidos y ácidos nucleicos codificantes. Esta estrategia combinada con ecografías supera los problemas de accesibilidad de los tejidos cartilaginosos a los que se enfrentaban los fármacos tradicionales y mejora la penetrancia y la transfección de los tejidos. El trabajo será realizado por investigadores noveles que también adquirirán competencias transferibles para que desarrollen su trayectoria profesional en este campo.

Objetivo

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.

Coordinador

UNIVERSITAIR MEDISCH CENTRUM UTRECHT
Aportación neta de la UEn
€ 265 619,88
Dirección
HEIDELBERGLAAN 100
3584 CX Utrecht
Países Bajos

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Región
West-Nederland Utrecht Utrecht
Tipo de actividad
Higher or Secondary Education Establishments
Enlaces
Coste total
€ 265 619,88

Participantes (11)