Descripción del proyecto
La terapia de células madre podría restaurar las redes neuronales encefálicas locales y distantes
La ciencia ha realizado grandes avances en cuanto a la comprensión de los circuitos neuronales del encéfalo sano y durante la enfermedad, así como en lo que respecta a su estructura y funcionamiento. Nuestros conocimientos sobre los circuitos encefálicos locales y distantes que se observan en las enfermedades neurodegenerativas, combinados con el potencial de las células madre embrionarias humanas para la regeneración celular y tisular, pueden hacer avanzar el tratamiento de patologías debilitantes como las enfermedades de Parkinson y de Huntington. El proyecto financiado con fondos europeos NSC-Reconstruct está ampliando los límites de la investigación sobre células madre para tipos de células individuales. En un ambicioso esfuerzo, el equipo prevé reconstruir redes locales mediante la incorporación de diversos tipos de células, así como restituir redes complejas y proyecciones corticales para una reparación totalmente funcional y eficaz en condiciones complicadas como en enfermedades neurodegenerativas y el ictus.
Objetivo
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.
Ámbito científico
Palabras clave
Programa(s)
Convocatoria de propuestas
Consulte otros proyectos de esta convocatoriaConvocatoria de subcontratación
H2020-SC1-2019-Single-Stage-RTD
Régimen de financiación
RIA - Research and Innovation actionCoordinador
20122 Milano
Italia