Engineering Minigenes as a Therapeutic Approach to Deliver Smaller Yet Functional Portion of Large Genes to the Retina that Exceed AAV Limited Cargo Capacity
Time: 11:30 am
day: Conference Day One
Details:
- Creating minigenes to overcome the challenges of packaging large genes into AAV
- Using minigenes to address unmet need in inherited retinal diseases
- Delaying photoreceptor degeneration in Leber Congenital Amaurosis using a minigene therapy for CEP290
- Developing a minigene therapy for Stargardt disease
