Gene therapy is the product of man's quest to eliminate diseases. Gene therapy has three
facets namely, gene silencing using siRNA, shRNA and miRNA, gene replacement where …

Abstract Aims Homozygous familial hypercholesterolaemia (HoFH) is a rare life-threatening
condition characterized by markedly elevated circulating levels of low-density lipoprotein …

Gene therapy is becoming an increasingly valuable tool to treat many genetic diseases with
no or limited treatment options. This is the case for hundreds of monogenic metabolic …

Over the years, various liver-derived in vitro model systems have been developed to enable
investigation of the potential adverse effects of chemicals and drugs. Liver tissue slices …

We describe the construction of a safe, replication-defective and efficient lentiviral vector
suitable for in vivo gene delivery. The reverse transcription of the vector was found to be a …

Liver transplantation is the accepted treatment for patients with acute liver failure and liver-
based metabolic disorders. However, donor organ shortage and lifelong need for …

Cardiovascular diseases remain a large global health problem. Although several
conventional small-molecule treatments are available for common cardiovascular problems …

Background: Familial hypercholesterolaemia (FH) affects approximately 1 in 500 people (10
million world-wide) and the elevated serum cholesterol concentrations lead to a more than …

The efficient delivery of therapeutic genes and appropriate gene expression are the crucial
issues for clinically relevant gene therapy. Viruses are naturally evolved vehicles which …

Hepatocyte transplantation has shown potential as an additional treatment modality for
certain diseases of the liver. To date, patients with liver-based metabolic disorders or acute …