Retinal gene therapy for Stargardt disease with dual AAV intein vectors is both safe and effective in large animal models AAV-intein-based gene therapy for Stargardt disease proves to be safe and efficacious in large animal models.

🔬 AAV-based gene therapy for Stargardt disease is safe and effective in large animal models

A work of #IvanaTrapani and #AAVantgardeBio !!!

📄 #ScienceAdvances | doi.org/10.1126/sciadv.adt9354

#GeneTherapy #Ophthalmology #AAV #Stargardt #VisionResearch