In Vivo Model Development for Amyotrophic Lateral Sclerosis

Protheragen offers a comprehensive in vivo animal model development service for Amyotrophic Lateral Sclerosis (ALS), supporting preclinical research and therapeutic development with a robust portfolio of validated models. Our service encompasses model selection, customization, and full experimental execution, providing clients with reliable, translationally relevant data to accelerate ALS research and drug discovery.

Animal models are indispensable tools in ALS research, enabling the study of disease mechanisms, biomarker discovery, and the evaluation of potential therapeutics in a physiological context. Protheragen utilizes a wide array of species—including Caenorhabditis elegans (worms), Drosophila melanogaster (fruit flies), Danio rerio (zebrafish), Mus musculus (mice), Rattus norvegicus (rats), and Macaca fascicularis (Cynomolgus monkeys)—each offering unique advantages for modeling human ALS. Our models incorporate key genetic mutations (such as SOD1, TARDBP, FUS, C9orf72, and VAPB) and disease-relevant manipulations, recapitulating the pathophysiological and clinical features of ALS. This diversity enables us to address a broad spectrum of scientific questions, from mechanistic studies to translational efficacy assessments.

Genetic Models

Genetic models are established by introducing disease-relevant mutations or transgenes (e.g., SOD1, TARDBP, FUS, C9orf72, VAPB, ATXN2) into various animal species, including mice, rats, zebrafish, worms, and flies. Methods include transgenic, knockin, knockout, and conditional genetic manipulations. These models closely mimic the genetic and molecular underpinnings of familial and sporadic ALS, allowing for the study of disease onset, progression, and pathophysiology. Key advantages include high construct validity, reproducibility, and the ability to model specific patient genotypes. Primary applications involve therapeutic efficacy testing, biomarker validation, mechanistic studies, and gene-environment interaction research.

Chemically-Induced Models

Chemically-induced models are generated by administering neurotoxic agents (such as cyclopiazonic acid, beta-sitosterol glucoside, or L-BMAA hydrochloride) to rodents or other species to provoke ALS-like neurodegeneration. This approach enables rapid induction of motor neuron pathology and disease phenotypes. Advantages include simplicity, cost-effectiveness, and the ability to study environmental contributors to ALS. These models are particularly useful for screening neuroprotective compounds, investigating toxin-mediated mechanisms, and exploring environmental risk factors.

Xenograft and Humanized Models

Xenograft and humanized models involve the transplantation of human-derived cells, tissues, or cerebrospinal fluid into rodents, or the genetic humanization of key ALS-linked genes. For example, xenografting fibroblasts expressing mutant human SOD1 into rat spinal cords or administering ALS patient CSF to mice. These models provide a unique platform for studying human-specific disease processes and testing cell-based or personalized therapies. Key advantages are the high translational relevance and the ability to evaluate human-targeted interventions. Applications include cell therapy assessment, evaluation of human disease mechanisms, and biomarker translation.

Protheragen delivers an end-to-end ALS animal model service, including model selection and generation, study design, dosing and administration, behavioral and functional assessments, tissue collection, and data analysis. Our efficacy endpoints encompass survival analysis, motor function tests (rotarod, grip strength, gait analysis), neuromuscular pathology (histology, immunohistochemistry), molecular biomarker quantification (qPCR, ELISA, Western blot), and neuroinflammation assays. We offer advanced analytical capabilities such as high-content imaging, transcriptomics, and proteomics. Rigorous quality control is maintained through standardized protocols, genetic verification, health monitoring, and comprehensive data validation, ensuring robust and reproducible results.

By partnering with Protheragen, you gain access to a scientifically rigorous, customizable, and fully supported ALS in vivo model platform. Our breadth of expertise, commitment to quality, and seamless project management empower your research from early discovery to preclinical validation. Contact us today to discuss how our ALS animal model services can accelerate your therapeutic development and advance your scientific goals.