With the advancement of genetic engineering and its integration into medicine, genetic analyses have increasingly been used for accurate disease diagnosis. At the same time, gene therapy has become one of the most promising areas in the medical field, offering potential one-time cures for genetic disorders.
What is Gene Therapy?
Gene therapy aims to restore the function of a specific gene responsible for a disease or syndrome. This is done by administering a drug that contains a genetically repaired version of the defective gene. The answer to “What is gene therapy?” is that it’s a technology used to treat genetic disorders by correcting or replacing faulty genes.
FDA-Approved Gene Therapy Treatments
Recently, the FDA approved gene therapy treatments for two conditions:
Retinal Pigment Epithelium Degeneration Type 20 (caused by mutations in the RPE65 gene)
Acute Myeloid Leukemia
Retinal pigment degeneration is a hereditary cause of blindness that gradually impairs central vision in both children and adults. The condition is currently treated using Luxturna, a gene therapy drug that contains a functional copy of the RPE65 gene. It is considered a one-time treatment that restores visual function.
Acute Myeloid Leukemia
Acute myeloid leukemia (AML) is a form of blood cancer commonly seen in children. The gene therapy drug Kymriah is used as a last-resort, one-time treatment when the patient does not respond to chemotherapy or other conventional therapies. In this method, the patient’s own T-cells are extracted, genetically modified to recognize and destroy cancer cells, and then reinfused back into the patient.
The Future of Gene Therapy
Patients often ask, “What is gene therapy?” because it offers real hope for treating genetic diseases. In our clinic, we perform genetic testing and consultations for diagnosing both of these conditions. Gene therapy continues to evolve as a powerful tool with the potential to revolutionize the treatment of inherited disorders, opening up entirely new horizons in medicine.