Gene Therapy Explained

Gene Therapy Overview

Gene therapy is a medical approach that provides an opportunity to treat inherited genetic diseases. There are more than 10,000 rare diseases, and most are caused by harmful changes to a single gene. Scientists have identified the genes responsible for many of these illnesses. Gene therapy aims to treat disease by introducing new genetic material to correct a faulty gene. Often, the therapy provides instructions for making a beneficial protein. Some well-known diseases attributable to changes in a single gene include cystic fibrosis, hemophilia, muscular dystrophy, spinal muscular atrophy, and sickle cell anemia. In addition, scientists are designing gene therapies to combat cancer and infectious diseases.

Gene Therapy Applications

DNA is the genetic material that we inherit from our parents, and its unique code inscribes a master plan for all our biological processes. DNA contains information for all the proteins produced in our body. Each gene carries instructions for creating a particular protein, and proteins carry out many cellular functions. There are five distinct strategies deployed in gene therapy including:

• Gene Addition supplements a cell with either a working copy of the mutated gene or a new gene.
• Gene Editing attempts to repair the original defective gene.
• Gene Silencing prevents protein production stemming from the detrimental gene.
• Reprogramming delivers one or more genes to completely change the cell.
• Cell Elimination delivers “suicide” genes to destroy the cell.

Gene Therapy Administration

Gene therapy can be administered directly to a patient. A specialized transporter is required to administer gene therapies because cells do not efficiently take up genes. Scientists are developing both viral and non-viral vectors for this purpose. Viral vectors are designed using a modified virus whose illness-causing elements have been removed and replaced with the therapeutic gene. Once the viral vector infects the cells, the therapeutic payload gets to work restoring normal physiology. Non-viral vectors are designed using synthetic molecules that protect the therapeutic gene and permit entry into cells. Once inside, the genetic cargo initiates salutary changes.