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 …

Kidney disease is a leading cause of morbidity and mortality across the globe. Current
interventions for kidney disease include dialysis and renal transplantation, which have …

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

Chronic obstructive pulmonary disease (COPD) is an age and smoking related progressive,
pulmonary disorder presenting with poorly reversible airflow limitation as a result of chronic …

Helper-dependent adenoviral vectors (HDAd) are deleted of all viral genes and they can
efficiently transduce a wide variety of dividing and non-dividing cells to mediate high …

The identification of> 100 genes causing inherited retinal degeneration and the promising
results of recent gene augmentation trials have led to an increase in the number of studies …

The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-
associated protein (Cas) 9 system is now widely used as a genome editing tool. CRISPR …

The emergence and re-emergence of pathogens with pandemic potential has been a
persistent issue throughout history. Recent decades have seen significant outbreaks of …

Current research highlights the transformative potential of gene therapy for PKD. Ongoing
studies are crucial to overcoming existing challenges and translating these findings into …

The application of cancer gene therapy has heretofore been restricted to local, or
locoregional, neoplastic disease contexts. This is owing to the lack of gene transfer vectors …