Phenylketonuria (PKU) or hyperphenylalaninemia is considered a paradigm for an inherited
(metabolic) liver defect and is, based on murine models that replicate all human pathology …

Solid lipid nanoparticles (SLNs) and nanostructured lipid carriers (NLCs) have been
recognized, among the large number of non-viral vectors for gene transfection, as an …

Objective Laron syndrome (LS) is a rare, autosomal recessive disorder in humans caused
by loss-of-function mutations of the growth hormone receptor (GHR) gene. To establish a …

In vertebrates, the liver is the central metabolic organ of the body, which carries out an
estimated 500 functions that range from general detoxification to protein synthesis, bile …

The principle of hydrodynamic delivery was initially used to develop a method for the
delivery of plasmids into mouse hepatocytes through tail vein injection and has been …

Hepatic gene therapy by delivering non-integrating therapeutic vectors in newborns remains
challenging due to the risk of dilution and loss of efficacy in the growing liver. Previously we …

Since its discovery, the Clustered Regularly Interspaced Short Palindromic Repeat (the
CRISPR) system has been increasingly applied to therapeutic genome editing. Employment …

Limited duration of transgene expression, insertional mutagenesis, and size limitations for
transgene cassettes pose challenges and risk factors for many gene therapy vectors. Here …

Development of an safe and efficient in vivo gene delivery method is indispensable for
molecular biology research and the progress in the following gene therapy. Over the past …

Hydrodynamic tail vein injection (HTV) is the" gold standard" for delivering naked DNA
vectors to mouse liver, thereby transfecting predominately perivenous hepatocytes. While …