Gene Therapy Cargoes Based on Viral Vector Delivery

Abstract

Viral vectors have proven useful in a broad spectrum of gene therapy applications due to their possibility to accommodate foreign genetic material for both local and systemic delivery. The wide range of viral vectors has enabled gene therapy applications for both acute and chronic diseases. Cancer gene therapy has been addressed by delivery of viral vectors expressing anti-tumor, toxic, and suicide genes for destruction of tumors. Delivery if immunostimulatory genes such as cytokines and chemokines has also been applied for cancer therapy. Moreover, oncolytic viruses specifically replicating in and killing tumor cells have been used as such for tumor eradication or in combination with tumor killing or immunostimulatory genes. In a broad meaning, vaccines against infectious diseases and various cancers can be considered as gene therapy, which has been highly successful not the least for development of effective COVID-19 vaccines. Viral vector-based gene therapy has also demonstrated encouraging and promising results for chronic diseases such as severe combined immunodeficiency (SCID), muscular dystrophy, and hemophilia. Preclinical gene therapy studies in animal models have demonstrated proof-of-concept for a wide range of disease indications. Clinical evaluation of drugs and vaccines in humans has showed high safety levels, good tolerance, and therapeutic efficacy. Several gene therapy drugs such as the adenovirus-based drug Gendicine® for non-small-cell lung cancer, the reovirus-based drug Reolysin® for ovarian cancer, lentivirus-based treatment of SCID-X1 disease, and the rhabdovirus-based vaccine Ervebo against Ebola virus disease, and adenovirus-based vaccines against COVID-19 have been developed.

Viral Vectors in Gene Therapy: Where Do We Stand in 2023?

“…Moreover, infectious diseases have been mainly subjected to vaccine development, which in a broad sense can be considered as gene therapy. As these areas have previously been described in detail elsewhere [ 250 ], only a short summary is included here ( Table 5 ).…”

Viral Vectors Engineered for Gene Therapy

“…In this review, the focus will be solely on viral vectors engineered for gene therapy. Previously, extensive reviews have described in detail the use of various viral vectors for a number of indications [8,9]. To avoid repetitiveness, different viral vectors are presented only briefly.…”