The model was able to predict optimized GFP sequences that had as many ... for the viral capsid of adeno-associated virus (AAV), a viral vector that is commonly used to deliver DNA.

“If you look at the number of vector genomes you're delivering to an individual ... and compared delivery efficiencies of the GFP molecules across the brain. AAV-PHP.B showed at least 40-fold higher ...

Giulio Cossu, MD, speaks to the lingering safety concerns related to ex vivo gene therapy in Duchenne muscular dystrophy (DMD) as long-term data are yet to be established.