Gene Therapy Research: What Is It and How Does It Work?

Gene Therapy Research: What Is It and How Does It Work?

Gene therapy, often described as gene editing, is the process of treating or preventing disease by inserting genetic material into a patient’s cells. Genetic material may come from modified viruses or bacteria (called vectors) that have been altered to contain healthy DNA that can replace a patient’s defective gene. Learn more about this new technology in this blog post!

What is gene therapy research?

Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat diseases by inserting genes into a patient’s cells, rather than using drugs or surgery.

If you’re interested in learning more about gene therapy research, read on for the current state of the field in what Cyagen has to offer.

How does it work?

Gene therapy is the insertion of genetic material into a person’s cells and tissues to treat diseases. New genetic material can be inserted in several ways. One way is to use genetically engineered viruses to carry new genes into cells. Viruses enter cells and insert new genes into DNA. The cell can then make a protein from the new gene.

Another way to insert new genetic material is to inject it directly into cells. This method is used to treat cancer because cancer cells grow rapidly and are easily reached with needles. Once inside the cell, new genes can be turned on or off, depending on the protein they encode.

A third way to insert new genetic material is to modify stem cells outside the body and then transplant them back into the patient. Stem cells are immature cells that can become any other type of cell in the body. The method is used to treat diseases that affect blood cells, such as leukemia and sickle cell anemia.

A fourth method of inserting new genetic material is called “in vivo” gene therapy. In this approach, the therapeutic gene is delivered directly to the target tissue using a virus or other delivery vehicle. This method is still experimental and has so far only been used in animal studies.


Gene therapy is an exciting area of ​​research with great promise for the future. Cyagen has accumulated a large amount of biological information and gene editing data over the last ten years as a comprehensive solution provider, as well as project experience in virus packaging, stable cell transfection, and gene editing model animals. We must be Your dependable supplier.

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