New ALS models provide hope for advancing research

New ALS models provide hope for advancing research

Amyotrophic Lateral Sclerosis, ALS,?also known as progressive frostbite, is the third most prevalent neurodegenerative disease in the world. It is caused by the death of motor neurons in the brain and spinal cord. The World Health Organization lists ALS as one of the top incurable diseases.?There are about 500,000 people suffering from ALS worldwide.

GemPharmatech’s ALS models

In recent years, numerous ALS-related genes have been identified, including dozens of reported mutations, such as C9ORF72, SOD1, FUS and TDP-43.?In view of the pathogenic genes and drug research hotspots related to ALS,?GemPharmatech focused on?SOD1,?TDP43?and?FUS, and constructed a number of?ALS related mouse models?including?B6-hSOD1 G93A,?hTDP43 A315T, and?hFUS R521C. These strains can be used to study the pharmacological effect of ALS therapeutics and pathogenesis of ALS.?

Preliminary validation data show the?hTDP43 A315T mouse model?simulates the pathological and behavioral characteristics of patients with ALS.

Other ALS-related models

Target Gene

Strain number

Strain name

RNF220?

T054274 B6-hRNF220

SOD1?

T055223 B6-Tg(hSOD1 G93A,hSOD1)

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