Full functional recovery has been achieved in an entire muscle using AAV vector-mediated gene therapy in a limb girdle muscular dystrophy hamster model, which has a mutation in the 1.2-kb gene for ...
Improved AAV tropism is essential for gene therapy to reach its full potential ... says Song. “Because the mechanism by which AAVs are targeted to specific cells is not fully understood ...
an adeno-associated virus (AAV) gene therapy candidate designed to deliver the gene for APOE2. APOE2 is thought to have a protective effect, as people expressing the gene tend to have a ...
Gene therapy can effectively ... into a powerful gene delivery mechanism. When two halves of a large therapeutic gene are encoded into adeno-associated virus (AAV) vectors, which are the most ...
The patient died from complications linked to a rare hyperinflammatory syndrome associated with exposure to high doses of the AAV therapy delivery vehicle.