The use of exosomes in clinical settings is progressively becoming a reality, as clinical trials
testing exosomes for diagnostic and therapeutic applications are generating remarkable …

Inappropriate stimulation or defective negative regulation of the type I interferon response
can lead to autoinflammation. In genetically uncharacterized cases of the type I …

This review aims to describe the past history, present techniques and future directions in
transcatheter treatment of patent ductus arteriosus (PDA). Transcatheter PDA closure is the …

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-associated …

Background Niemann-Pick disease type C (NP-C) is a rare neurovisceral lysosomal lipid
storage disease characterized by progressive neurological deterioration. Published data on the …

Introduction Mitochondrial fatty acid β‐oxidation defects (FAODs) are a group of severe
inherited metabolic diseases, most of which can be treated with favorable prognosis following …

Exosomes are a subset of extracellular vesicles essential for cell–cell communication in
health and disease with the ability to transport nucleic acids, functional proteins and other …

Urea cycle defects (UCDs) are severe inherited metabolic diseases with high unmet needs
which present a permanent risk of hyperammonaemic decompensation and subsequent …

The urea cycle is a liver-based pathway enabling disposal of nitrogen waste. Urea cycle
disorders (UCDs) are inherited metabolic diseases caused by deficiency of enzymes or …

Recombinant adeno-associated virus (rAAV) vectors are a promising platform for in vivo
gene therapy. The presence of neutralizing antibodies (Nab) against AAV capsids decreases …