Inherited metabolic diseases of the liver are characterized by deficiency of a hepatic enzyme
or protein often resulting in life‐threatening disease. The remaining liver function is usually …

Liver transplantation is an important therapeutic option for many individuals with metabolic
liver disease. Nevertheless, the invasive nature of surgery and limitations of donor organ …

Adeno‐associated virus (AAV) vectors are ideal for performing gene repair due to their
ability to target multiple different genomic loci, low immunogenicity, capability to achieve …

Background & Aims Wilson's disease (WD) is an autosomal recessive disorder of copper
metabolism caused by loss-of-function mutations in ATP7B, which encodes a copper …

To achieve permanent correction of Wilson's disease by a cell therapy approach,
replacement of diseased hepatocytes with healthy hepatocytes is desirable. There is a …

The toxic milk (tx) mouse is a rodent model for Wilson disease, an inherited disorder of
copper overload. Here we assessed the effect of copper accumulation in the tx mouse on …

Recombinant adeno-associated virus (rAAV) vectors have clear potential for use in gene
targeting but low correction efficiencies remain the primary drawback. One approach to …

Wilson disease (WD) is caused by ATPase copper-transporting beta (ATP7B) mutations and
results in copper toxicity in liver and brain. Although the defective gene was identified in …

Wilson's disease (WD) is a rare genetic disorder inherited as an autosomal recessive trait.
The signs and symptoms of this disease are related to dysfunctional ATP7B protein which …

Adipose-derived stem cells (ADSCs) derived from adipose tissue have the capacity to
differentiate into endodermal, mesoderm, and ectodermal cell lineages in vitro, which are an …