IMPROVING THE EFFECTIVENESS AND SAFETY OF EPIGENETIC EDITING IN BRAIN REGENERATION

The central nervous system has a limited capacity for self-repair.

Therefore, technologies to replace lost neurons after an injury, including stroke or neurodegenerative diseases, are of great need.

The project REGENERAR aims to develop a non-viral delivery formulation up to TRL4 for the delivery of an epigenetic reprogramming formulation to reprogram glial cells into neurons.

The in vitro safety and targeting of the formulation will be tested against glial cells and organotypic cortical slice cultures. Several routes of administrations will be tested to enhance the accumulation of the formulation in the brain. In addition, the in vivo safety, elimination, targeting and epigenetic reprogramming will be evaluated.

In addition, toxicological studies will be performed in GLP conditions to evaluate both in vivo systemic and local (brain) effects of the formulation.

The development of the non-viral formulation will take in account the contributes of key stakeholders in the area of gene editing, health institutions, patient associations, among others. A roadmap will be developed for upscaling innovation to higher TRLs upon the end of the action.