Ex vivo CRISPR/Cas9‐mediated gene editing in hepatocytes using homology‐directed repair
(HDR) is a potential alternative curative therapy to organ transplantation for metabolic liver …

Background Autologous hepatocyte transplantation after ex vivo gene therapy is an alternative
to liver transplantation for metabolic liver disease. Here we evaluate ex vivo gene therapy …

Conventional therapy for hereditary tyrosinemia type-1 (HT1) with 2-(2-nitro-4-trifluoromethylbenzoyl)−1,3-cyclohexanedione
(NTBC) delays and in some cases fails to prevent disease …

The effectiveness of cell-based therapies to treat liver failure is often limited by the diseased
liver environment. Here, we provide preclinical proof of concept for hepatocyte …

The effectiveness of cell-based therapies to treat liver failure is limited by the diseased liver
environment. Herein we provide preclinical proof-of-concept for the treatment of liver failure …

Methods We performed laparoscopic partial hepatectomies on 6-week-old (15-20 kg)
fumarylacetoacetate hydrolase (FAH)-deficient pigs (n= 6) and isolated primary hepatocytes ex vivo…

Hereditary tyrosinemia type 1 (HT1) is an autosomal recessive disorder caused by
deficiency of fumarylacetoacetate hydrolase (FAH). It has been previously shown that ex vivo …

Orthotopic liver transplantation remains the only curative therapy for inborn errors of
metabolism. Given the tremendous success for primary immunodeficiencies using ex-vivo gene …