To review the viral vector-based gene therapy landscape, we focus on these three highly regarded vector platforms and describe mechanisms of action and their roles in treating human disease. However, despite these successes, many challenges still limit these approaches from attaining their full potential. They have led the way in preclinical and clinical successes in the past two decades. At present, the three key vector strategies are based on adenoviruses, adeno-associated viruses, and lentiviruses. These modalities range from vector-based cancer therapies, to treating monogenic diseases with life-altering outcomes. Within the past 5 years, the gene therapy field has seen a wave of drugs based on viral vectors that have gained regulatory approval that come in a variety of designs and purposes. At the heart of this dichotomous field are the viral-based vectors, the delivery vehicles that have allowed researchers and clinicians to develop powerful drug platforms, and have radically changed the face of medicine. Treatment of patients with this class of investigational drugs has resulted in severe adverse effects and, even in rare cases, death.
It has seen great strides in combating human disease, has given hope to patients and families with limited treatment options, but has also been subject to many setbacks. Throughout its 40-year history, the field of gene therapy has been marked by many transitions.
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