Definition
Viral vector gene therapy is a medical technique that uses modified viruses to deliver genetic material into a patient's cells to treat or prevent disease. This approach leverages the natural ability of viruses to enter cells, allowing for the introduction of therapeutic genes.
Etymology
The term "viral vector" combines "viral," relating to viruses, with "vector," a term from biology indicating a carrier that transfers genetic material. "Gene therapy" describes the treatment of disease by modifying a person's genes.
Example usage
'Researchers are exploring viral vector gene therapy as a promising treatment for inherited genetic disorders.'
Interesting fact
One of the first successful applications of viral vector gene therapy was in 1990, when it was used to treat a four-year-old girl with severe combined immunodeficiency (SCID). This landmark case paved the way for further advancements in the field.
Source: National Human Genome Research Institute
Explanation and detail
How it works
Viral vector gene therapy involves using viruses as vehicles to deliver therapeutic genes to a patient's cells. The viruses are modified to remove their ability to cause disease. Instead, they carry healthy genes into cells, where these genes can produce the necessary proteins to correct genetic disorders.
Types of viral vectors
Common viral vectors include adenoviruses, adeno-associated viruses (AAV), lentiviruses, and retroviruses. Each type has unique properties that make it suitable for specific therapeutic applications. For instance, AAV is often used due to its low immunogenicity and ability to infect non-dividing cells.
Applications
Viral vector gene therapy holds promise for a range of genetic disorders, including cystic fibrosis, hemophilia, and certain types of inherited blindness. It is also being explored for use in cancer treatment, where it can be employed to deliver genes that either directly kill cancer cells or stimulate the immune system to attack them.
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