Viral vectors for gene therapy

This chapter provides a general background on viral vectors used for gene therapy applications and then more specifically focuses on the recent translational progress that has been made with adeno-associated virus (AAV) vectors. Viral vectors have emerged as powerful delivery tools for the enhancement, reduction, and restoration of gene expression. Retrovirus, lentivirus, adenovirus, herpes simplex virus, and AAV, have been used for ex vivo and in vivo gene transfer. The attributes and limitations of these vectors are discussed as well as their translational successes and failures. Due to its many recent clinical advances, the use of AAV in current clinical trials is listed and categorized by targeted organ (i.e., eye, liver, central nervous system, and others), and the predominant serotype capsids and administration routes utilized for each particular target organ are summarized. This chapter also covers AAV manufacturing, including contemporary platforms for large-scale vector production and quality control measures to ensure safety and efficacy.