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Human Amyotrophic Lateral Sclerosis iPSC-Derived Models
Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's Disease, is a devastating neurodegenerative condition characterized by the progressive loss of motor neurons, leading to debilitating muscle weakness and paralysis. Despite its severity, ALS remains without a cure, necessitating innovative approaches for therapeutic development.
Understanding ALS
ALS manifests in two primary forms: familial ALS (fALS), which affects approximately 10% of patients and has a genetic basis, and sporadic ALS, which comprises the majority of cases. Both forms share clinical characteristics and present significant challenges in diagnosis and treatment.
The Need for Advanced Models
Developing effective therapies for ALS is exceptionally challenging, with a high attrition rate in clinical trials. To address this, advanced and validated disease models are crucial for accurately studying ALS pathogenesis and screening potential treatments.
Our Approach
At NeuroProof, we employ a rigorous assay validation strategy encompassing constructed, face, and predictive validation methods to ensure the reliability and relevance of our models.
Our primary focus is on the use of human-induced pluripotent stem cell (iPSC)-derived spinal motor neurons, offering a versatile platform for modeling ALS pathology and conducting phenotypic screening.
Key Mutations and Mechanisms:
Our iPSC-derived motor neurons include mutations associated with familial ALS, such as C9orf72, SOD1, and TDP43, which cover a spectrum of pathological mechanisms implicated in ALS progression.
These mutations contribute to various aspects of ALS pathology, including protein misfolding, oxidative stress, mitochondrial dysfunction, and excitotoxicity, providing valuable insights into disease mechanisms.
Hyperexcitation Assays
Utilizing electrophysiological MEA recordings, our hyperexcitation assays mimic pathological neuronal activity observed in ALS, offering a valuable tool for disease modeling and drug screening.
We demonstrate disturbed excitatory-inhibitory balance in ALS mutant cell lines, highlighting their relevance for studying disease mechanisms and evaluating therapeutic interventions.
Comprehensive Screening Services
Our assays encompass a range of readouts, including molecular markers such as TDP-43 accumulation, cell viability assays, and imaging techniques, providing a holistic approach to ALS research.
Customers can customize their screening protocols, including standalone MEA readouts or combinations with other assays, tailored to their specific research needs.
Continuous Innovation
NeuroProof is committed to expanding our assay portfolio to address emerging challenges in ALS research, including the development of new cell lines and co-culture systems with astrocytes and microglia.
From sourcing diseased cell lines to compound screening campaigns, we offer integrated project solutions to accelerate ALS drug discovery efforts.
Contact Us
For further information on our ALS iPSC model and assay services, or to discuss collaboration opportunities, please contact us.