Safety and efficacy analysis of in vivo lentiviral gene therapy in pre-clinical ARC syndrome models - PubMed
4 hours ago
- #liver disease
- #gene therapy
- #VPS33B
- Study evaluates safety and efficacy of liver-targeted lentiviral gene therapy for ARC syndrome, caused by VPS33B defects.
- Liver-specific lentiviral vector combined with macrophage depletion improves survival, growth, liver function, reduces fibrosis, and restores bile canaliculi in a mouse model.
- The treatment shows a favorable safety profile and supports targeted gene therapy as a promising approach for ARC syndrome and similar liver diseases.