What is Gene Therapy?
Gene therapy is an experimental treatment that can either add a new gene, or repair a damaged one within cells to help prevent or treat certain diseases. It may also train the immune system to recognize and attack cancer cells or shield healthy cells from chemotherapy treatments.
When a genetic defect or mutation occurs in the DNA of a gene, it makes it difficult for the body to produce the protein coded for by that gene. This condition is known as genetic deformation and may be hereditary or acquired through environmental exposures like radiation.
In addition to replacing missing or non-functioning genes, researchers are investigating ways to repair defective ones through editing. This can be accomplished through a process known as base editing, in which DNA is cut with an enzyme that alters one strand’s bases; naturally, cells then repair themselves naturally after this change has taken place.
Scientists typically use a delivery vehicle (vector) to introduce a new gene. These vectors can be plasmids, nanostructured materials or viruses.
Viral vectors are the best way to introduce genetic material into cells, as they can enter cells and deliver therapeutic DNA or RNA. Common viral vectors include retroviruses and adeno-associated viruses.
However, these viral vectors can present risks such as toxicity, inflammatory reactions and issues targeting DNA or RNA molecules. These concerns could potentially stall progress in gene therapy research. Scientists are working hard to develop safer and more effective vectors that don’t trigger an immune response.