In Vivo Model Development for Mitochondrial Disease

Protheragen offers comprehensive in vivo animal model development services for Mitochondrial Disease, supporting the advancement of therapeutic research and drug discovery. Our specialized models enable researchers to investigate disease mechanisms, evaluate candidate treatments, and accelerate translational studies with high fidelity and reproducibility.

Mitochondrial Diseases encompass a diverse group of disorders characterized by impaired mitochondrial function, often resulting in multi-systemic clinical manifestations. Accurate animal models are essential for elucidating disease pathogenesis and assessing therapeutic efficacy. Protheragen utilizes a range of species and strains, including Danio rerio (zebrafish) and various Mus musculus (mouse) strains such as C57BL/6, CD-1, Kunming, and mt-Keima. These models were selected for their genetic tractability and physiological relevance, offering robust platforms that recapitulate key aspects of human mitochondrial dysfunction and pathophysiology.

Genetic Knockout Models

Genetic knockout models are established by targeted deletion or disruption of specific mitochondrial genes, such as dld in zebrafish or Ndufs4 in mice. These models reliably mimic inherited mitochondrial disorders and allow for the investigation of gene-specific disease mechanisms. Key advantages include precise genetic control, stable phenotype transmission, and high relevance to human monogenic mitochondrial diseases. They are primarily used for studying disease etiology, evaluating gene-targeted therapies, and exploring compensatory metabolic pathways.

Chemically-Induced Models

Chemically-induced models are generated by administering compounds that selectively impair mitochondrial function, such as 3-nitropropionic acid, paracetamol, or lipopolysaccharide in various mouse strains. This approach enables the rapid induction of mitochondrial dysfunction and related pathology. The main advantages are ease of induction, dose-dependent effects, and suitability for high-throughput screening. These models are widely used to assess drug toxicity, investigate metabolic resilience, and evaluate protective interventions against mitochondrial damage.

Environmentally-Induced Models

Environmentally-induced models involve exposing animals to external stressors, such as cigarette smoke, to induce mitochondrial impairment. For example, the mt-Keima mouse strain is utilized to monitor mitochondrial turnover and mitophagy in response to environmental insults. These models closely mimic complex environmental contributions to mitochondrial disease and allow for the assessment of lifestyle and exposure-related risk factors. They are particularly valuable for studying disease-modifying interventions and understanding the interplay between genetics and environment.

Protheragen delivers a complete solution for in vivo Mitochondrial Disease model development, from model selection and customization to experimental execution and data analysis. Key efficacy endpoints include assessment of mitochondrial function (respiratory chain activity, ATP production), clinical scoring, histopathological evaluation, behavioral and cognitive assays, and survival analysis. Our analytical capabilities encompass molecular, biochemical, and imaging techniques such as qPCR, Western blotting, immunohistochemistry, and live-cell fluorescence imaging. Rigorous quality control measures are integrated at every stage, including genetic validation, environmental monitoring, and standardized protocols to ensure reproducibility and data integrity.

Partnering with Protheragen provides access to scientifically validated animal models, expert technical support, and a seamless workflow tailored to your research objectives. Our commitment to quality, innovation, and collaboration ensures that your mitochondrial disease studies are both impactful and efficient. Contact us today to discuss your project needs and accelerate your mitochondrial therapeutics development.