Placental stem cell correction of murine intermediate maple syrup urine disease
Kristen J. Skvorak, Kenneth Dorko, Fabio Marongiu, Veysel Tahan, Marc C. Hansel, Roberto Gramignoli, K. Michael Gibson, Stephen C. Strom – 23 November 2012 – There is improved survival and partial metabolic correction of a mouse intermediate maple syrup urine disease (iMSUD) model after allogenic hepatocyte transplantation, confirming that a small number of enzyme‐proficient liver‐engrafted cells can improve phenotype. However, clinical shortages of suitable livers for hepatocyte isolation indicate a need for alternative cell sources.