achieve up to 1,000-fold higher RNA expression in the brain and 100-fold higher RNA expression in the spinal cord of NHPs relative to AAV9, which is the naturally occurring AAV serotype that is ...
The failure to demonstrate efficacy of epidural steroid injections for lumbar spinal stenosis suggests that this extremely common practice does not stand on firm evidence. Furthermore, some ...
Spinal muscular atrophy (SMA ... of their approach in cell lines before delivering the treatment via an AAV viral vector into a mouse model of SMA. They found that the treatment resulted in ...