CORDIS Project
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HEPAGENE focuses on understanding liver cell diversity to improve gene therapies for metabolic diseases. It aims to identify how different liver cell types contribute to liver growth and their potential for effective gene modification.
In vivo genetic engineering of hepatocytes may represent a definitive cure for monogenic metabolic diseases.
Integration of the therapeutic transgene into the target cell genome is essential for long-term expression after a single dose early in life and can be achieved by semi-randomly integrating lentiviral vectors or site-specific genome editing.
Maintenance of the genetic modification upon hepatocytes proliferation in liver growth and turnover requires targeting the cells underlying these pro…
UNIVERSITA VITA-SALUTE SAN RAFFAELE
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