Treating ALS by Stopping Faulty Proteins

Neurodegenerative disorders are diseases that cause a progressive loss of nerve cells; that can result in devastating problems with movement or brain function. ALS or Amyotrophic lateral sclerosis is one such disease, impacting neurons that control voluntary muscles. While the majority of cases happen seemingly at random, about ten percent are due to errors in genes. ALS has been associated with mutations in over ten genes so far.

Scientists are hopeful that advances in technologies that manipulate gene expression will offer new treatment avenues for these debilitating diseases. In the case of ALS, scientists are working on developing a molecule called an antisense oligonucleotide. If the molecule works as intended, it stops a gene from being made into a protein. In patients, it could prevent the production of a harmful protein and hopefully, halt the progression of the disease.