CORDIS Project
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This project focuses on improving gene therapy for neurological disorders by developing targeted delivery systems for therapeutic genes into the central nervous system. It aims to enhance the efficiency of adeno-associated virus vectors while minimizing off-target effects, particularly in the liver, using innovative st…
Targeting therapeutic genes selectively into the central nervous system (CNS) is a crucial precondition for translation of gene therapy strategies into human trials.
The current multidisciplinary proposal integrates expertise identified as essential in the effective acceleration of research to overcome bottlenecks in the field including: 1) Inefficiency of therapy delivery to the CNS because of factors like the blood-brain barrier (BBB); 2) Poor understanding of disease mechanisms at the molecul…
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