eBook | Viral and non-viral gene delivery approaches

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Gene therapies have revolutionized modern medicine by offering the potential to treat and cure diseases at their genetic root. Traditionally, these therapies have relied on viral vectors – including lentiviruses, adenoviruses and adeno-associated viruses (AAVs) – to deliver therapeutic genetic cargo to target cells and tissues. These viral delivery systems have demonstrated remarkable efficiency in transducing cells and enabling efficient gene expression, making them the most widely used approach in early gene therapy development.

However, viral vectors present several significant limitations, such as cytotoxicity, limited cargo capacity and integration risks that have warranted the exploration of alternative approaches. In response to these challenges, non-viral approaches have emerged as promising alternatives. For example, exosomes and nanotechnology present innovative delivery platforms that aim to overcome the limitations of viral vectors.

This eBook explores the evolving landscape of gene therapy delivery systems, examining both the established viral methodologies and the emerging non-viral technologies that are shaping the future of gene therapy.