Gene therapy is a common word used in the biopharmaceutical and medical field microbiologists. You might have encountered several articles involving this term. What does it mean, and why is gene therapy paving the way to a new future?
What is gene therapy?
It is a technique that uses genes to treat diseases. Billions of dollars are being put into this field as it will allow doctors to insert genes in humans to cure diseases. It is an effective alternative to drugs and surgery, making the treatment easier, quicker, and cheaper.
How does gene therapy work?
Direct insertion of genes inside a human body is ineffective. Gene therapy requires insertion, removal, or modification of certain disease-causing genes through specific carriers called vectors. Healthier genes are introduced in the body that targets the malfunctioned genes, hence curing the human body. The DNA is carefully selected, and the disease-causing parts are removed or replaced.
Virus vectors and their role in gene therapy
Viruses can infect cells directly; for example, a notorious family of viruses called lentivirus is known to cause deadly diseases like HIV. Lentivirus has the unique ability to infect both dividing and non-dividing cells due to which lentiviral vectors are high in demand. They are being used to treat a variety of diseases in the present, including prostate cancer, vascular transplants, chronic granulomatous diseases, hemophilia A, rheumatoid arthritis, and diabetes mellitus.
Virus vectors are simply carriers that can break through the nucleus. The unique thing about them is that they have their viral gene removed, and a genetically modified gene is inserted into them. The virus often used for this purpose is called the Adeno-Associated Virus, also known as AAV, which can easily infect humans but is not known to cause any diseases.
These engineered virus particles have a modified genome, and they can insert their genome into the host’s cells. The genetically modified DNA is delivered into the nucleus without affecting the chromosome.
Virus production: The future drug industry
The gene therapy research is going at a breakneck pace, and it will be replacing the drugs used today. Gene therapy is believed to change the human race. The whole process demands high-end viruses produced in a controlled environment. Virus production requires GMP systems for quality standards and consistency. If the quality is not maintained, it can cause massive damage to people. Only a few companies can live up to these standards and are producing world-class virus vectors.
After researching the virus vectors and gene therapy in-depth, looking for virus vectors providers has now become easier. Some companies give you a choice to create a vector-based on your specific research needs. One of them is VectorBuilder, an online platform where you can place your order and get your vector delivered. They are experts in lentiviral and Adeno-associated virus vectors manufacturing along with cloning, vector packaging, and vector design with the GMP system you can find on their official website.
