Public & Patient Series – Liver directed lentiviral gene therapy for inherited cholestatic disorders
Approximately 1 in 2500 neonates develop cholestatic jaundice, often due to rare genetic disorders with no cure and severe morbidity. This talk will explore how innovative lentiviral gene therapies could transform treatment, using ARC syndrome as a case study. We will highlight key breakthroughs in optimizing gene delivery and ensuring vector safety— crucial steps toward…
Free
