Gene Therapy
Medical conceptAbout
Gene therapy is a medical approach that modifies genes to treat or prevent diseases. It involves altering the genetic code to correct the underlying cause of a disorder, rather than relying on traditional treatments like medication or surgery. Gene therapy can replace a faulty gene with a healthy one, inactivate a disease-causing gene, or introduce a new gene to help the body fight a disease. Techniques include gene transfer and genome editing, such as CRISPR-Cas9, which can modify existing DNA. Gene therapy uses vectors, often modified viruses, to deliver genetic material into cells. It can be applied in vivo (directly into the body) or ex vivo (outside the body, with cells later returned to the patient). While promising, gene therapy is still largely in clinical trials, with ongoing research for conditions like cancer, genetic disorders, and HIV/AIDS. The FDA has approved several gene therapies, highlighting its potential as a groundbreaking treatment. Despite challenges, gene therapy offers new possibilities for treating genetic diseases by directly addressing their root causes.
