Gene therapy is a rapidly evolving field that offers the potential to cure or treat various diseases by targeting the underlying genetic causes . It involves introducing genetic material into a person’s cells to replace or repair a defective gene, thereby restoring normal function This approach has shown promise in the treatment of vision loss, cancer, and other serious and rare diseases .
How Gene Therapy Works
Gene therapy works by delivering genetic material into a person’s cells to correct or replace a defective gene Sometimes, a person is born with a defective or missing gene, which is referred to as a genetically inherited mutation In other cases, healthy genes can mutate over the course of a person’s life due to environmental exposures .
The genes in our cells provide the information that determines the functions of different cells, tissues, and organs in our bodies . By targeting and correcting specific genes, gene therapy aims to restore normal cellular function and ultimately improve the overall health of the individual .
Potential Benefits of Gene Therapy
Gene therapy offers several potential benefits in the field of medicine. It has the potential to cure or treat diseases that were previously considered incurable or difficult to manage . By targeting the underlying genetic causes of diseases, gene therapy has the potential to provide long-term solutions and improve the quality of life for patients .
In addition, gene therapy has the potential to prevent diseases in individuals who have genes predisposing them to certain conditions . If gene therapy is performed on reproductive cells, it could prevent the transmission of unfavorable genetic diseases and disorders to future generations .
Progress and Approvals in Gene Therapy
The field of gene therapy has seen significant progress in recent years, with the development of groundbreaking treatments that have the potential to save lives . Regulatory bodies, such as the U.S. Food and Drug Administration (FDA), are committed to supporting the development of gene therapy products for unmet medical needs .
The FDA has approved innovative gene therapies to treat a wide range of diseases, including spinal muscular atrophy, a rare disease and leading genetic cause of infant mortality . These approvals mark significant milestones in the transformational power of gene and cell therapies .