Engineering Gene Therapy: Advances and Barriers

Currently, 33 gene-therapy drugs/products have been approved in the clinic. Over 3000 completed and ongoing clinical gene therapy trials reported worldwide. The development/maturation of tools for manipulation delivery, as well molecular advances diagnosis genetic diseases, played a central role therapy, which greatly revolutionized field. Versatile diverse manipulations deliveries, with possibility short long-term effects, are vital advantages tools, paving road development new therapies. However, efficacy safety concerns, immune system responses, laborious approaches developing manufacturing, unknown drug interactions host high cost drugs/products, significant barriers therapy. Here, authors review attempts engineering that undertaken last three decades used focusing on 1) gene-editing platforms, 2) viral delivery systems, 3) nonviral tools. In this comprehensive review, principles these their application modern therapies discussed. Furthermore, trends future directions