The NIH National Institute of Neurological Disorders and Stroke (NINDS) awarded University of Illinois bioengineering professors Thomas Gaj (BSD) and Pablo Perez-Pinera (ACPP) a five-year, approximately $4.8 million translational research grant to develop an optimized gene therapy for amyotrophic lateral sclerosis (ALS), a severely debilitating and fatal neurological disorder. 

A single genetic mutation can have profound consequences, as demonstrated in neurodegenerative diseases such as amyotrophic lateral sclerosis or Huntington’s disease. A new study by University of Illinois Urbana-Champaign researchers used a targeted CRISPR technique in the central nervous systems of mice to turn off production of mutant proteins that can cause ALS and Huntington’s disease.

With a new CRISPR gene-editing methodology, scientists from the University of Illinois at Urbana-Champaign inactivated one of the genes responsible for an inherited form of amyotrophic lateral sclerosis – a debilitating and fatal neurological disease for which there is no cure. The novel treatment slowed disease progression, improved muscle function and extended lifespan in mice with an aggressive form of ALS.