ALS Drug Discovery Studies

Amyotrophic Lateral Sclerosis (ALS) is a neurodegenerative disease that is characterized by stiff muscles, muscle twitching, and gradual muscle atrophy resulting in difficulty speaking, swallowing, and eventually breathing. About 90% of ALS cases have no known etiology, while the remaining 10% have a genetic cause. Mutations in over 20 genes have been associated with familial ALS, with mutations in 4 genes accounting for the majority of familial cases – SOD1, FUS, TDP-43, and C9orf72. Charles River offers an extensive portfolio of in vivo models and in vitro assays for Amyotrophic Lateral Sclerosis Studies. 

Image of person looking at resources on a computer with a cup of coffee. Charles River has many resources for your Amyotrophic Lateral Sclerosis research

Amyotrophic Lateral Sclerosis Resources

Check out our Amyotrophic Lateral Sclerosis posters, publications and other scientific resources…

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In Vitro Assays For Amyotrophic Lateral Sclerosis (ALS)

Charles River has established an in vitro model from cultures of human induced pluripotent stem cells (hiPSCs) from patient cell lines harboring a mutation in one of the 4 genes which account for the majority of familial ALS. We have optimized a robust motor neuron differentiation protocol that is amenable to high-throughput screening. Using this protocol, approximately 80-85% of the differentiated neurons express mature motor neuron markers. This physiologically relevant cell system combined with a scalable differentiation protocol, allows for high-throughput screening of small molecule libraries or functional genomics-type approaches (RNAi / CRISPR-Cas9), using high content imaging or biomarker readouts. 

Molecular phenotypes associated with ALS - Neurite outgrowth in MNs.

In Vitro Assays with human induced pluripotent stem cells (hiPSCs) for Amyotrophic Lateral Sclerosis (ALS)

Disease relevant in vitro assays for Amyotrophic Lateral Sclerosis in motor neurons derived from control and patient iPSCs Learn more about Charles river in vitro assays for ALS studies.

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In Vivo Models for Amyotrophic Lateral Sclerosis (ALS)

Charles River offers multiple disease relevant in vivo pharmacology models to support your Amyotrophic Lateral Sclerosis (ALS) research. We have a validated in vivo transgenic SOD1-G93A model. The transgenic SOD1 (superoxide dismutase 1) mice express a G93A mutant form of human SOD1. SOD1 mice (TgN-SOD1-G93A-1Gur) exhibit a phenotype similar to amyotrophic lateral sclerosis (ALS) in humans.

Study Endpoints

  • Basic Monitoring: Body weight, Kaplan-Meier survival plot, Disease onset and clinical scoring.
  • Behavioral Testing: Gait, balance, and posture changes using fine motor kinematic analysis, Rotarod tests, Grip strength, Open field tests and Rearing frequency.
  • Imaging: MRI to detect neurodegeneration in the brain stem and spinal cord, MRS to assess metabolic changes in the brain stem, FDG-PET imaging to assess metabolic changes.

Validation Data

Click below to see a sample data set.


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