Preclinical Mouse Models for Reliable ALS Therapeutic Insights
Amyotrophic Lateral Sclerosis (ALS), often known as Lou Gehrig's disease, is the third most prevalent neurodegenerative disorder worldwide. It relentlessly targets motor neurons in the brain and spinal cord, leading to progressive muscle weakness, paralysis, and ultimately, respiratory failure. For the hundreds of thousands of patients and families it affects globally, every new research breakthrough brings new hope for finding a cure.
Why Reliable Animal Models Remain Indispensable for New Research
Every revolutionary therapy like Tofersen, first requires robust preclinical research to bridge the gap from concept to clinic. Without appropriate animal models, we cannot systematically evaluate the efficacy and safety of novel treatments.
To decipher the pathogenesis of ALS and accelerate related drug discovery, GemPharmatech has developed two pivotal ALS mouse models, B6-hSOD1 G93A, hSOD1, and B6-hTDP43wtxA315T, utilizing gene editing technology. These models can serve as invaluable tools for the scientific community to explore new therapeutic targets.
A Data-Driven Window into Disease Progression
The B6-hSOD1 G93A, hSOD1 mouse model carries the full-length humanized SOD1 gene with the G93A mutation, making it exceptionally well-suited for evaluating SOD1-targeting ASO drugs like Tofersen and other therapeutic strategies.
This model exhibits clear and quantifiable disease progression, providing critical readouts for efficacy studies:
At 3 months of age, male mice begin to show evident muscle atrophy.
A progressive decline in motor ability begins at 5-5.5 months of age.
By testing whether a therapeutic candidate can delay the onset or mitigate the severity of these milestones, researchers can efficiently identify the most promising treatments.
Your Partner in Precision ALS Research
At GemPharmatech, we believe defeating a complex disease such as ALS requires intricate models complex as the disease itself. These genetically engineered mouse models are validated and reliable partners in your research, delivering the clear data you need to move from hypothesis to groundbreaking discovery.
Don't let model variability cloud your results. Choose a platform that provides the precision and predictability your vital work deserves.
Explore the detailed specifications and data sheets of our ALS models to see how they can illuminate the path for your next breakthrough.