Amyotrophic lateral sclerosis, more commonly known as ALS, is a progressive neurodegenerative disease in the central nervous system that impacts the brain and spinal cord, resulting in a loss of muscle control.
It is colloquially referred to as Lou Gehrig’s Disease, after the famous baseball player who battled it in the late 1930s, and does not yet have a cure. To better understand the language of ALS, the ALS Glossary below provides definitions for research terms commonly used by our Innovation Ecosystem and the extended ALS community.
recombinant AAV (rAAV) production
The creation of adeno-associated viruses vectors that can infect cells but not damage them, for gene therapy.
A technique that can examine the quantity and sequences of RNA in a sample. It analyzes the transcriptome, which shows which genes encoded in DNA are turned on or off and to what extent