The world population is suffering a lot of genetic diseases. These diseases have been very hard to tackle so far. Fortunately, since a few decades viral vectors and gene therapies became more and more a widely used approach to genetically engineer disorders, develop vaccines, and study the function of genes and proteins. This includes the increasing rate of clinical trials where these techniques are adapted. This article provides insights about what there viral vectors are, and how this technique is used in drug development.
Viral vectors are used in gene therapy, a treatment that aims to correct or compensate for genetic disorders by introducing healthy copies of genes into a person's cells. Some examples of genetic disorders that have been treated or are being tested in clinical trials using viral vectors include:
Hemophilia: a disorder in which the blood does not clot properly, causing excessive bleeding.
Cystic fibrosis: a disorder that affects the lungs and other organs, causing them to become clogged with thick mucus.
Leber's congenital amaurosis: a disorder that affects the retina, causing severe vision loss in infants.
In addition to gene therapy, viral vectors have also been used to develop cancer therapies. These therapies involve using viral vectors to deliver genes that can kill cancer cells or make the immune system attack cancer cells.
Viral vectors are also being used for vaccine development. For example, the COVID-19 vaccines developed by Pfizer-BioNTech and Moderna are based on messenger RNA (mRNA), which is a genetic code for the spike protein found on the surface of the SARS-CoV-2 virus and delivered to cells by a viral vector to instruct the cells to produce the spike protein and trigger an immune response.
It's important to note that while viral vectors offer a lot of promise as a tool for therapy, the field is still in its early stages, and more research is needed to understand their potential and address any safety concerns fully.
How do viral vectors work?
Imagine you have an essential package to deliver to a specific location. But the address is hard to find and the package is too big to carry alone. That's where viral vectors come in - they're like delivery trucks that can help you get the package to the right place.
In the same way, scientists use viral vectors to deliver healthy copies of genes to cells that are not working correctly. These genes act like instructions to help the cells function better. So, for example, if a person has a disease caused by a missing or damaged gene, scientists can use a viral vector to deliver a working copy of that gene to their cells to fix the disease.
Scientists use these viral vectors to test new drugs by delivering the drug to the cells with the viral vector. If the drug works in the cells, scientists may test it in animal models, and if it looks promising, then in human clinical trials, like how you would test a package before sending it to the final destination.
How do they help?
One way viral vectors contribute to drug development is by enabling gene therapy. Gene therapy involves inserting functional genes into cells to replace or correct missing or defective genes that cause disease. Viral vectors can be used to deliver therapeutic genes to target cells, and once inside the cells, the genes can produce functional proteins or regulate gene expression.
Viral vectors can also be used to create vaccines. Vaccines introduce a harmless part of the pathogen into the body to stimulate an immune response. Viral vectors can be engineered to carry genes that encode parts of the pathogen, which can then be used as a vaccine to stimulate an immune response without causing disease.
Furthermore, viral vectors can be used to study the function of genes and proteins. Scientists can use viral vectors to express or suppress specific genes in cells or animals, which can help them understand the role of these genes in various biological processes and diseases. This information can be used to identify new drug targets and develop more effective treatments for diseases.
Overall, viral vectors are becoming increasingly important in creating new patient therapies.
