-
Categories
-
Pharmaceutical Intermediates
-
Active Pharmaceutical Ingredients
-
Food Additives
- Industrial Coatings
- Agrochemicals
- Dyes and Pigments
- Surfactant
- Flavors and Fragrances
- Chemical Reagents
- Catalyst and Auxiliary
- Natural Products
- Inorganic Chemistry
-
Organic Chemistry
-
Biochemical Engineering
- Analytical Chemistry
-
Cosmetic Ingredient
- Water Treatment Chemical
-
Pharmaceutical Intermediates
Promotion
ECHEMI Mall
Wholesale
Weekly Price
Exhibition
News
-
Trade Service
Summary
Fusion sarcoma (FUS) is an RNA-binding protein genetically and pathologically associated with the rare forms of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD)
main
Amyotrophic lateral sclerosis (ALS) is a fatal neurological disease characterized primarily by degeneration of the spinal cord cortex, spinal bulb, and spinal motor neurons (MNs), resulting in paralysis and death
Mutations in FUS are associated with the most aggressive, early-onset forms of ALS5,6 and the rarer FTD7,8
To establish a faithful ALS-FUS model to explore disease mechanisms, identify therapeutic targets, and test therapeutic candidates, we generated a series of FUS knock-in mouse lines in which als mutagenesis directly targets internal Derived mouse FUS locus
Finally, we found that an experimental ASO targeting the FUS transcript (ION363) efficiently silenced wild-type and mutant FUS in the brain and spinal cord of P517L and Δ14 heterozygous mice
