By Philip CohenBIGGER, stronger muscles can be produced by gene therapy, an experiment on mice suggests. Loss of muscle mass can cripple and kill patients with conditions such as muscular dystrophy. And even ordinary ageing saps up to one third of our strength. “Slowing down that process could mean the difference between someone being mobile […]
“Overview of AAV
Adeno-Associated Virus (AAV) as a Vector for Gene Therapy
AAV was discovered over 50 years ago and has since become one of the leading gene delivery vectors in clinical development. As a result of its unique biology, simple structure, and no known disease associations, AAV could become the vector of choice for most gene therapy applications. Gene therapy using rAAV has been demonstrated to be safe and well-tolerated in virtually every clinical setting in which it has been used. These studies, along with basic research on its biology, have revealed many facets of this vector that can be applied to future efforts.