Description du projet
La thérapie aux cellules souches pourrait permettre de restaurer les réseaux neuronaux proches et distants du cerveau
La science a fait des progrès considérables dans la compréhension des circuits neuronaux du cerveau, à la fois dans un contexte de santé et de maladie, mais également en termes de structures et de fonctions. Notre connaissance du circuit cérébral proche et distant observable dans les cas de maladies neurodégénératives, combinée au potentiel des cellules souches embryonnaires humaines pour la régénération des cellules et des tissus, permettent des avancées dans le traitement de pathologies débilitantes comme les maladies de Parkinson et de Huntington. Le projet NSC-Reconstruct financé par l’UE repousse les limites de la recherche en matière de cellules souches pour les types de cellules uniques. L’équipe a pour objectif ambitieux de reconstruire les réseaux proches en incorporant de nombreux types de cellules, et de restaurer les réseaux complexes et les projections corticales, afin de véritablement réparer de manière fonctionnelle les pathologies complexes telles que les maladies neurodégénératives et les AVC.
Objectif
Neurodegenerative diseases and brain injuries affect large patient groups and carry large unmet clinical needs. NSC-Reconstruct will respond to these needs by developing innovative therapies based on cell replacement, cell reprogramming and circuit reconstruction that have the potential to transform how we treat a wide range of neurological diseases and disorders.
In this area of clinical science European research has generated groundbreaking knowledge that has resulted in a pioneering human embryonic stem cell-derived product that is now entering clinical trials for Parkinson’s Disease (PD). In NSC-Reconstruct we will move beyond the replacement of a single neuronal type towards future cell therapies with enhanced authenticity, functions and compositions.
NSC-Reconstruct will address PD as an example of disorder for which single neuron replacement has been developed. Our focus in PD will be on incorporating improved cell types and on local network reconstruction. We will also work on repairing long distance networks such as those affected in Huntington’s disease and finally in restoring the complex networks and projections of the cerebral cortex to achieve effective repair in conditions known to affect this structure, such as trauma or stroke.
NSC-Reconstruct will lead to (i) the generation of neuronal types with clinically relevant functionality starting from human pluripotent stem cells or through in situ reprogramming, (ii) the optimization of donor cell composition through grafting of functional modules comprising distinct cell types capable of forming appropriate connections, (iii) the restoration of function through the reconstruction of these damaged connections and finally (iv) a greater understanding of the immunogenicity of grafted cells and how this can be minimized.
Ultimately, NSC-Reconstruct will provide unique knowledge and products that will pave the way for future CNS cell replacement therapies using functionally enhanced and immune-tolerant cells.
Champ scientifique
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RIA - Research and Innovation actionCoordinateur
20122 Milano
Italie