Controlled Muscle-Specific Gene Delivery
Treatment with the AAV gene therapy with doxycycline (B) induced expression of the target gene.
Adeno-associated virus (AAV) vector-based gene therapy has the potential to treat a number of rare diseases. However, only two AAV-based gene therapies have been approved by the FDA, primarily due to toxicity and lack of specificity.
Inventor(s): Renping Zhou
Keywords(s): Gene editing
Category(s): Technology Classifications > Gene Therapy, Technology Classifications > Healthcare & Life Sciences