Award
National Center for Advancing Translational Sciences 5U01TR005355-02
Personalized prime editing as a platform for hepatic inborn errors of metabolism
Recipient
Not specified
Award Amount
$2,710,325.00
Ceiling
$2,710,325.00
Awarded
August 06, 2025
Identifier
5U01TR005355-02
NIH grant awarded to develop personalized prime editing therapies for hepatic inborn errors of metabolism using advanced gene editing technologies.
Description
This is a NIH grant project focusing on developing personalized prime editing therapies for hepatic inborn errors of metabolism, including phenylketonuria (PKU), urea cycle disorders, and organic acidemias. The project involves advanced gene editing technologies, regulatory innovation, and a platform approach for multiple rare diseases.