REGENERAR: Improving the Effectiveness and Safety of Epigenetic Editing in Brain Regeneration

Overview

Project Summary

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. This formulation will be subjected to several safety tests against glial cells and advanced in vitro models such as organotypic cultures of brain cortical slices. In vivo toxicological studies, in accordance with good laboratory practices, will evaluate the systemic and local (in the brain) effects of the formulation. Various routes of administration will be tested with the aim of increasing the bioaccumulation of the formulation in the brain. In addition to investigating the biodistribution and elimination from the organism, the safety and efficacy of the formulation will be evaluated regarding its capacity for targeted delivery to glial cells and epigenetic reprogramming. The development of the non-viral formulation will count on the contribution of national and European health entities and patient associations, in order to create a strategy for the future translation of this innovative technology into clinical practice.

Main Goals

Proof-of-concept demonstration of non-constitutive brain epigenetic reprogramming using a non-viral delivery system targeting astrocytes.

Project Details