What can result from defective genes being replaced through gene therapy?

Gene therapy aims to treat or prevent diseases by modifying genes involved in the disease process. When defective genes are replaced or repaired through this therapeutic approach, it can lead to significant improvements in health, and in some cases, a complete cure for certain genetic disorders. This is especially relevant in conditions caused by single-gene defects, such as some types of inherited blindness, muscular dystrophy, and cystic fibrosis.

The underlying principle is that by correcting the genetic defect, normal cellular functions can be restored, leading to the alleviation of symptoms or reversal of disease progression. As a result, patients may experience better health outcomes, enhanced quality of life, and in some situations, a complete resolution of their condition. Therefore, the successful implementation of gene therapy has the potential to transform the management of many diseases, offering hope for individuals suffering from previously untreatable genetic disorders.