By Payal Khandelwal, PhD
The number of gene therapy–based treatments has grown significantly since they first appeared nearly three decades ago. This has created profound optimism about our potential to develop a cure for diseases such as cancer and AIDS. One of the most effective contributors to the success of gene therapy is the ability to use viruses as vehicles for delivering genes to their targets. Initially, murine retroviruses were recruited for this purpose, but more recently, adenovirus (Ad) and adeno-associated virus (AAV) have become the vehicles of choice. In fact, Ad and AAV studies account for over 25% of all ongoing gene therapy trials. However, producing sufficient quantities of pure clinical-grade virus, which is required to ensure biosafety, is not an easy task.
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