Potent in vivo gene editing in skeletal muscle of non-human primates by a novel, ultracompact CRISPR system delivered via a single AAV vector.

Researchers are enhancing the best features of AAV capsids and overcoming their limitations to accelerate gene-based therapies.

Adeno-associated viruses (AAV) have emerged as the lead vector in clinical trials ... The tolerogenic environment in the liver can also stimulate the production of regulatory T-cells (Tregs), which ...

Figure 2: Activity of eGFP and β-glucuronidase in sections of murine liver after intravenous injection ... Figure 4: Distribution and activity of GFP and β-glucuronidase in brain.