New discoveries and technological developments in healthcare have always been pushing the limits of what was previously thought to be achievable. The application of viral vectors to gene therapy and genome editing is a prime example of such a revolutionary step forward. Since their discovery in the 1980s, our knowledge of and ability to use viral vectors have advanced significantly. In this post, we’ll talk about the revival of viral vectors in medicine, their use in gene therapy and genetic editing, and how they’re changing the face of healthcare forever.
Where Did Viral Vectors Come From?
To introduce desired genetic material into host cells, scientists have developed viral vectors. Researchers have known for a long time that viruses might be used as a delivery mechanism due to their capacity to penetrate cells and modify their DNA. The first studies in this area were conducted in the 1980s, and they focused on creating gene treatments for diseases including severe combined immunodeficiency (SCID) and cystic fibrosis. Unfortunately, early clinical trials ran into several problems, including as immune response concerns, low efficacy, and even severe adverse effects. Thus, the viral vector technology was largely ignored for several years as a means of carrying out gene therapy.
The Revival of Virus Carriers
Researchers never lost faith in the potential of viral vectors despite the difficulties they encountered at the outset. Safer and more effective viral vectors have been developed thanks to recent developments in genetic engineering and a deeper understanding of viral biology. The comeback of viral vectors has changed the face of medicine by facilitating advances in areas like gene therapy and genetic editing.
Adeno-associated viruses (AAVs) and lentiviruses are two of the most promising viral vectors. AAVs are appealing for gene therapy because they are non-pathogenic and can transport genes to many different types of cells. Conversely, lentiviruses inherit their ability to integrate their genetic material into the host cell’s DNA from their retrovirus ancestors. Because of this quality, they are excellent candidates for permanent gene expression and modification.
Potential Use of Viruses as Delivery Vehicles for Gene Therapy
Gene therapies for a wide range of disorders have made significant progress thanks to the use of viral vectors. The goal of gene therapy is to correct or eliminate a genetic condition by introducing healthy copies of the affected gene into the affected tissue or organ. Many therapies, including those for inherited blindness, spinal muscular atrophy, and even some types of cancer, have been approved thanks to the effective use of viral vectors in gene therapy.
The approval of voretigene neparvovec (Luxturna), an AAV-based treatment for a rare form of inherited blindness, is one of the most significant successes in gene therapy to date. The exceptional effectiveness of this therapy in recovering patients’ vision shows the promise of viral vectors in healing otherwise incurable disorders.
Vector-borne diseases and genome editing
Advances in genetic editing, especially with the development of CRISPR-Cas9 technology, have also been made possible by the return of viral vectors. This game-changing technology allows researchers to make specific modifications to the DNA of live organisms, which has far-reaching medicinal implications.
Delivering CRISPR-Cas9 components into target cells via lentiviral vectors has been widely employed to facilitate genome editing for research and possibly therapeutic uses. Preclinical investigations have shown that they are effective at repairing genetic abnormalities that cause disorders like sickle cell anemia and beta-thalassemia.
Patients with a wide variety of genetic abnormalities and chronic diseases have reason to be hopeful thanks to recent developments in gene therapy and genetic editing made possible by the revival of viral vectors in healthcare. We may anticipate the development of even more cutting-edge treatments in the coming years as our knowledge of viral vectors and their applications expands.