Viral Vectors in Gene Therapy and Clinical Applications

Abstract

Developments in gene therapy, coupled with advances in genome sequencing and a greater understanding of DNA sequences, have given rise to an exciting area of research. The use of viral vectors in gene therapy has become a very promising and fast-emerging technology over the past few decades. Despite previous setbacks, the approval of viral vector therapies worldwide, with many in late-stage clinical trials has led to a significant increase in research in this area of gene therapy. Retroviral, adenoviral, adeno-associated viral, and lentiviral vectors are all key vectors currently being researched and used in clinical trials. There are many challenges with the use of viral vectors that are yet to be overcome including cost of production, the immune response, and the ability to precisely regulate the expression of the transgene. However, with increased numbers of clinical trials showing efficacy, safety, and growing financial investment, the future use of viral vectors in gene therapy is increasingly promising.