Gene Therapy and its Impact on ALS

Melia Harp


ALS, also known as amyotrophic lateral sclerosis, is a disease that damages brain cells and can cause a loss of function in the muscles (2). This disease is lethal and currently does not have a cure, but modern science and medicine might be able to change that with the help of gene therapy (2). One of the genes directly involved with ALS and gene therapy are the gene C9orf72 (4).  These gene either have a loss of function or becomes toxic (4). A recent study has been done where they test gene therapy. Gene therapy works by producing a protein called oligonucleotides that can attempt to help save the dying cells (2). Gene therapy is a fairly new field that is being tested. Although there is a long way to go, the future in gene therapy looks very bright based on the trials that have been ran already. The progress that has recently been made in gene therapy is exciting and could change the future of treatment for not only ALS but many other diseases like cancer and other neurodegenerative diseases. 

Full Text:



  • There are currently no refbacks.