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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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target modulation

Also referred to as “gene modulation” or “target gene modulation,” it refers to the process of temporarily altering gene expression, including the silencing of a gene function or increasing expression. The goal of target modulation is to provide some benefit or conform for those suffering from specific ailments.

target validation

Process of demonstrating that a molecular target is directly involved in a disease process, and that modulation of the target will likely have a therapeutic effect. Validations involve performing tests several times to check the same outcomes are always obtained, or by performing tests by a different research group to support outcomes.

TDP-43

A protein typically found within the cell’s nucleus. In ALS patients, it becomes dysfunctional and clumps (aggregates). TDP-43 buildup occurs in about 97% of people with ALS — including those with and without genetic mutations. Ongoing research is focused on TDP-43’s potential as an ALS biomarker.

transgene

A gene (or genetic material) that has been isolated from one organism and then transferred into another.

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