Advancements in Gene Therapy

 Advancements in Gene Therapy

Source: https://www.sciencenews.org/article/first-gene-therapy-muscular-dystrophy-kids

Additional Link: https://www.ncbi.nlm.nih.gov/books/NBK482346/#:~:text=Duchenne%20muscular%20dystrophy%20(DMD)%20is%20one%20of%20the%20most%20severe,muscle%20fiber%20degeneration%20and%20weakness.

Duchenne muscular dystrophy is a genetic disease that results in progressive muscular weakness that can cause fatality by the age of 25. This is because it is caused by mutations in the dystrophin gene, which normally makes a large protein that acts as a shock absorber to keep muscle cells intact and fatality is caused when heart muscles and the muscles that control breathing deteriorate. As of present day, this disease is incurable. 

The article, “The first gene therapy for muscular dystrophy has been approved for some kids” posted by ScienceNews stated that the first gene therapy for children with this disease was approved for the use of children between the ages of 4-5. The article states that the treatment produces , “A protein about one-third the size of the original protein. The shortened gene is packed into harmless viruses for delivery to muscle cells” (ScienceNews, 2023). This treatment is made to target the underlying source of the disease and prevent it from happening. 

It is very interesting to hear new advancements in medicine! Even though this is just a start for the treatment of this disease, I am excited to hear about the transformations this treatment can result in, and I hope it can help a lot of kids in the future.