Phenylketonuria (PKU) is an autosomal recessive inborn error of phenylalanine metabolism
caused by deficiency in the enzyme phenylalanine hydroxylase that converts phenylalanine …

More than 950 phenylalanine hydroxylase (PAH) gene variants have been identified in
people with phenylketonuria (PKU). These vary in their consequences for the residual level …

Prime editing is a highly versatile CRISPR-based genome editing technology that works
without DNA double-strand break formation. Despite rapid technological advances, in vivo …

CRISPR–Cas-based genome editing holds great promise for targeting genetic disorders,
including inborn errors of hepatocyte metabolism. Precise correction of disease-causing …

Phenylketonuria (PKU) is an autosomal recessive inborn error of metabolism caused by a
deficiency in the hepatic enzyme phenylalanine hydroxylase (PAH). If left untreated, the …

DNA base editors use deaminases fused to a programmable DNA-binding protein for
targeted nucleotide conversion. However, the most widely used TadA deaminases lack post …

The success of Onpattro™(patisiran) clearly demonstrates the utility of lipid nanoparticle
(LNP) systems for enabling gene therapies. These systems are composed of ionizable …

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 …

Over the last decade, pioneering liver-directed gene therapy trials for haemophilia B have
achieved sustained clinical improvement after a single systemic injection of adeno …

Introduction: Nucleic-acid-based biopharmaceuticals enclose a remarkable potential for
treating debilitating or life-threatening diseases that currently remain incurable. This …