![]() In most cases, the natural ability of viruses to enter cells and deliver genetic material is exploited to transfer therapeutic genes to target cells. Gene therapy aims at the transfer of therapeutic genes into a patient's cells to treat diseases on the genetic level. Over 70 years after the idea of gene therapy was put down into writing for the first time, research endeavors in viral vehicle development have come to fruition: at least 13 viral-based gene therapy products have been approved for the treatment of cancer or rare monogenic diseases to date. ![]() ![]() This review describes and discusses the recent trends in capsid and vector genome engineering, with particular emphasis on synthetic-biological approaches. It is anticipated that the emerging field of synthetic-biologically engineered AAV vectors can shape future gene therapeutic approaches and thus the design of tomorrow's gene delivery vectors. Concepts from synthetic biology enable the control and fine-tuning of vector function at different stages of cellular transduction and gene expression. Driven by the high clinical potential, a new generation of synthetic-biologically engineered AAV vectors is on the rise. This prospect of marking a new era in gene therapy has fostered both investigations of the fundamental AAV biology as well as engineering studies to enhance delivery vehicles. ![]() Pharmaceutical companies are investing in this small and nonpathogenic gene shuttle to increase the therapeutic portfolios within the coming years. Three recent approvals and over 100 ongoing clinical trials make adeno-associated virus (AAV)-based vectors the leading gene delivery vehicles in gene therapy. ![]()
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