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Target ALS

A New York based ALS research organization that's leading the fight for a cure.

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Glossary

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.

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recombinant AAV (rAAV) production

The creation of adeno-associated viruses vectors that can infect cells but not damage them, for gene therapy.

RNA-seq (RNA-sequencing)

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

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