Firstly, the viral vector. The company uses a non-replicating adeno-associated vector (AAV) to transport the genetic material to the heart cells. Particular serotypes of AAV are well known for ...

Robert Kotin and colleagues at the US National Heart, Lung, and Blood Institute first demonstrated its suitability for AAV manufacturing. They infected Sf9 cell lines — derived from the fall ...

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

NanoCas, an ultracompact nuclease, enables efficient muscle editing in NHPs using a single AAV - advancing gene editing for hard-to-target tissues.

Correctly measuring AAV vectors, as they are called, is critical to their safety and efficacy. In a recently released study, researchers at the National Institute of Standards and Technology ...