ERC Consolidator Grant · 2024
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 processes. Little is known about post-natal liver growth and how different hepatocyte subsets contribute to it. Unexpectedly, we found that most hepatocytes are quiescent during liver growth, and a…
From the public funding record at EU CORDIS. Describes the funded project, not the reviews below.