Description: Gene therapy is an innovative technique that aims to modify a person’s genes to treat or cure diseases. This methodology is based on the introduction, removal, or modification of genetic material within a patient’s cells. Gene therapy can be used to correct genetic defects that cause hereditary diseases, as well as to combat acquired diseases, such as certain types of cancer. Through different approaches, such as transferring healthy genes to replace defective ones or gene editing to correct mutations, this technique has the potential to provide solutions to conditions that were previously considered untreatable. The relevance of gene therapy lies in its ability to address the root causes of diseases at the molecular level, which could transform medical treatment and improve the quality of life for millions of people. As research advances, new strategies and technologies, such as CRISPR, are being developed that allow for more precise and efficient gene editing, thus expanding the possibilities for the application of gene therapy in modern medicine.
History: Gene therapy began to take shape in the 1970s when scientists started exploring the possibility of introducing genetic material into human cells. An important milestone occurred in 1990 when the first successful gene therapy was performed on a patient with severe combined immunodeficiency (SCID). Since then, gene therapy has evolved significantly, with advancements in gene delivery techniques and understanding of human genetics. In 2017, the FDA approved the first gene therapy treatment for a genetic disease, marking a turning point in medicine.
Uses: Gene therapy is primarily used to treat genetic diseases, such as muscular dystrophy, cystic fibrosis, and certain types of cancer. Its application is also being researched for viral diseases, such as HIV, and neurodegenerative conditions, such as Alzheimer’s disease. Additionally, gene therapies are being developed to improve eye health and treat metabolic disorders.
Examples: A notable example of gene therapy is the Zolgensma treatment, approved for spinal muscular atrophy, which uses a modified virus to deliver a functional gene to the patient’s cells. Another case is the Luxturna treatment, used to treat a hereditary form of blindness. Both treatments have proven effective in improving the quality of life for patients.
