Liver transplantation is the most effective treatment for end-stage liver disease. Transplant
indications have been progressively increasing, with a huge discrepancy between the …

Diseases that affect the liver account for approximately 2 million deaths worldwide each
year. The increasing prevalence of these diseases and the limited efficacy of current …

Hereditary tyrosinemia type 1 is an inborn error of amino acid metabolism characterized by
deficiency of fumarylacetoacetate hydrolase (FAH). Only limited treatment options (eg, oral …

Phenylketonuria (PKU) is the most common inborn error of metabolism of the liver, and
results from mutations of both alleles of the phenylalanine hydroxylase gene (PAH). As such …

Aromatic amino acids (AAA), including phenylalanine (Phe), tyrosine (Tyr), and tryptophan
(Trp), are essential building blocks of proteins, however, they have been implicated in …

Organ transplantation is the definitive treatment for end‐stage solid organ diseases, yet
biological and logistical barriers reduce the rate of successful organ transplants. As such …

Gene therapy has been used successfully in a diverse array of diseases, and, more recently,
this technique has gained interest in the field of organ transplantation. Biological and …

Background: Hereditary tyrosinemia type 1 is a rare genetic disorder leading to liver
cirrhosis and hepatocellular carcinoma. Few decades ago, dietary measures and ultimately …

The majority of monogenic liver diseases are autosomal recessive disorders, with few being
sex-related or co-dominant. Although orthotopic liver transplantation (LT) is currently the …

遗传性酪氨酸血症 Ⅰ 型 (HT-1) 是因延胡索酰乙酰乙酸水解酶缺陷所致的一种常染色体隐性
遗传代谢病. 患者体内酪氨酸分解代谢受阻, 毒性代谢物蓄积, 导致严重肝功能损害 …