When discussing challenges in gene therapy, delivery of the genetic payload is always at the top of the list. Whether discussing viral or nonviral gene delivery, challenges are present in every aspect ...
Current treatments focus on controlling inflammation and minimizing long-term damage. Learn more.
Adeno-associated viruses (AAV) have emerged as pivotal delivery vehicles for gene therapy due to their overall safety (less immunogenic), efficiency (broad tropism), and long-term gene expression.