Public Health Global Conference

Gene therapy refers to the emerging discipline of treating, and even potentially curing, disease caused by genetic disorders by modifying or replacing faulty genes within the cells of a patient. Thus, through these procedures including gene editing and gene replacement, gene therapy directly targets the roots of diseases and holds open the hope of treating conditions that were considered in the past to be incurable. Gene therapy innovations can boast of several achievements, starting with the cure for cystic fibrosis, hemophilia, and some other forms of inherited blindness. Indeed, these achievements will transform the care of patients and set boundaries in personalized medicine.

Gene editing with such technology as CRISPR-Cas9 now enables a modification of targeted genes directly in the right manner, allowing researchers to correct mutations that cause genetic disease. The approach does not create much damage to healthy cells, and control is underway for treatment outcomes. Gene editing can directly repair or disable defective genes so that problems such as muscular dystrophy or sickle cell anemia are corrected directly to greatly enhance the quality of life for patients.

Apart from correction, gene replacement therapy would introduce the functional, working genes to complement gaps resulting from the nonfunctional or missing ones. Such a strategy applies best for diseases resulting from the mutation of a single gene since the introduction of a healthy gene copy will improve the situation toward normal working values. Treatments developed through this process are thus being applied to treat rare genetic conditions that include SMA. Such an application is gaining momentum.

It is the mode of gene therapy delivery that transfers therapeutic genes to targeted cells without any adverse effect. Viral vectors are the classic gene delivery methods wherein therapeutic genes are transfected across the cell boundaries; newer research in non-viral gene delivery is also being studied for increasing safety and efficacy.

The gene therapy domain is moving at a very fast pace. Despite that, several ethical issues have to be addressed. Besides the cost and regulatory complexity, there is pretty strong progress in this area. It is an emerging therapeutic tool for genetic as well as chronic diseases. It is bringing a new era of precision medicine to the world.