New technology shows potential to improve potency and durability benefits in gene therapy
WATERTOWN, Mass. - Gene therapy has traditionally been conceptualized as a one-time, curative treatment option; however, research shows that there may be a need for subsequent doses years after initial treatment. While adeno-associated viral (AAV) vectors are a core part of this powerful therapeutic approach, they present two key challenges in gene therapy.
The first challenge is their immunogenicity. In gene therapy, the formation of neutralizing antibodies (Nabs) in response to AAV vector administration precludes retreatment of a patient due to the potentially dangerous immune response that would occur after a second or third administration of the therapy.
The second obstacle relates to their durability. AAV vectors ...









