Gene therapy has been approved as a treatment for which condition?

Gene therapy has been notably approved for the treatment of genetic conditions where the root cause is directly related to mutations in specific genes. Cystic fibrosis is primarily caused by mutations in the CFTR gene, which disrupts the production and function of a critical protein involved in salt transport in epithelial cells. The aim of gene therapy in this context is to deliver a functional copy of the CFTR gene to the patient's cells, thereby addressing the underlying genetic defect.

In contrast, while advancements are being made, gene therapy specifically for sickle cell anemia, diabetes, or multiple sclerosis may still be in research phases or have not yet received broad regulatory approval as a standard treatment. Sickle cell anemia treatments involving gene therapies exist but may not have received the same level of established approval that has been seen with cystic fibrosis. Diabetes, being primarily a metabolic disorder, and multiple sclerosis, an autoimmune disease, do not typically involve gene therapy as a core treatment currently approved.