Most of today’s gene therapies rely on viruses
This article details the case of 10-year-old Will Ungerer, who has Duchenne muscular dystrophy and received an experimental gene therapy at age 5 where functional gene copies are introduced in the patient’s cells to replace or supplement defective or absent genes. Due to this therapy, he had significant improvements in his physical abilities such as climbing stairs. Although gene therapy is effective there are many challenges such as safety concerns, delivery methods, and the high costs of treatment. “The FDA has approved seven other gene therapies for rare genetic diseases, all since 2017,” showing that ongoing clinical trials and regulatory processes make it hard for gene therapy to get approved. The second article discusses some potential benefits of gene therapy such as treating inherited disorders like cystic fibrosis and certain cancers, with some therapies already FDA-approved for conditions like Leber congenital amaurosis and blood cancers. However, they can also carry risks such as cancer and allergic reactions, leading to FDA approval for some gene transfer therapies and ongoing research into genome editing.
Gene therapy represents a significant breakthrough in medical science, offering hope for patients with genetic disorders like Duchenne muscular dystrophy. The ability of gene therapy to introduce functional gene copies directly addresses the root cause of many genetic diseases, leading to great improvements in patients suffering from physical disabilities. However, the challenges related to safety, delivery methods, and especially the high costs pose significant barriers to its widespread adoption and accessibility. The FDA's cautious approach in approving gene therapies, while ensuring patient safety, may slow the availability of these potentially life-altering treatments; however, those measures are necessary for the future of personalized medicine. I believe that more money should be invested in research aimed at improving gene therapy since every patient is different and the best treatment is one that is targeted to a patient’s specific genes.