Viral vectors for in vivo gene therapies with transfer of deoxyribonucleic acid
Bulgarian Medical Journal, 2025, 19(2), 23-29.
T. Vekov1, E. Becheva-Kraychir1,2, I. Gramatikov1
1 MHAT “Heart and Brain” – Pleven
2 Trakia University – Stara Zagora
Abstract. In vivo gene therapies based on the transfer of deoxyribonucleic acid (DNA) are commonly used for the treatment of a genetic disease, which affects a single specific gene, or when they are targeted to a target visceral organ. Modern gene therapy based on in vivo DNA transfer uses mainly viral vectors presenting genetically modified adenoviruses (Ad) or adeno-associated viruses (AAVs). With gene therapy in vivo, a functional transgene is introduced directly in human organism using a specific vector. Today, several drug products approved for use that represent in vivo gene therapies based on viral vector platforms are available.
Key words: viral vectors, in vivo gene therapies, deoxyribonucleic acid