commentary equal, or greater, clinical relevance. 5 Thus far, a majority of therapeutic agents proposed for treatment of neurodegenerative disease are secreted trophic factors such as insulin-like growth factor 1 and glial cell line–derived neurotrophic factor. 29 Because most trophic factors are typically secreted by glia, it is easy to envision that rAAV-mediated overexpres-sion of trophic fa...

Duchenne muscular dystrophy (BMD) is an inherited X-link disease. The incidence of this muscle-wasting disease is 1:5000 male live births. Mutation in the gene coding for dystrophin is the main cause of BMD. Most cases of this disease succumb to respiratory and cardiac failure in 3rd to 4th decades. The slow progression of BMD and recent achievement of gene therapies make it as an appropriate c...

Transient transfection of plasmids into suspension HEK293 cells is still the most commonly used method for production recombinant adeno-associated virus (rAAV) gene therapies – in this episode, Diane Golebiowski and Marissa Stanvick discuss ways to optimize that process.