CORDIS Project
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This project focuses on improving gene therapy using adeno-associated virus vectors by addressing immune responses that hinder their effectiveness. It aims to identify immune markers and test immunosuppressive regimens to enhance the safety and efficacy of gene transfer for treating genetic diseases.
Adeno-associated virus (AAV) vectors are among the most efficient vectors for in vivo gene transfer.
Experience in human gene transfer trials showed that targeting hepatocytes with AAV vectors has the potential to correct diseases like hemophilia, congenital blindness, and lipoprotein lipase deficiency.
However, these studies also suggest that one of the last obstacles to be overcome in order to achieve long-term disease correction is the immune system.
As a result of exposure to wild type AAV,…
UNIVERSITE PIERRE ET MARIE CURIE - PARIS 6
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