In Vivo Model Development for Complement-Mediated Disease

Protheragen offers a comprehensive in vivo animal model development service tailored for Complement-Mediated Disease research. Leveraging advanced genetic engineering and rigorous scientific methodology, our platform provides robust, reproducible, and clinically relevant mouse models to support the development and evaluation of novel therapeutics targeting the complement system.

Complement-Mediated Diseases encompass a broad spectrum of disorders driven by dysregulation of the complement system, a critical component of innate immunity. Accurate animal models are essential for unraveling disease mechanisms and assessing therapeutic efficacy. Protheragen utilizes Mus musculus (mouse) as the primary species due to its genetic tractability and physiological similarities to humans. We employ a variety of strains, including C57BL/6 (B6) and NOD, as well as specialized models such as transgenic (C3, C5, C7), knockout (C7, Cfh), and immune-deficient (SCID) lines. These models closely recapitulate human complement deficiencies and dysregulation, providing invaluable insights into disease pathogenesis and drug response.

Genetic Models

Genetic models utilize precise genetic modifications—such as gene knockouts, knock-ins, and transgenic expression—to mimic specific complement deficiencies or overactivation states observed in human diseases. For example, Protheragen offers C3 transgenic, C5 transgenic (on a SCID background), C7 knockout/transgenic, and Cfh knockout mice. These models are generated using state-of-the-art gene editing technologies and are ideal for studying the role of individual complement components, dissecting disease pathways, and evaluating the efficacy and safety of complement-targeted therapeutics. Key advantages include high specificity, reproducibility, and the ability to model rare or complex genetic backgrounds.

Immune-Deficient Models

Immune-deficient models, such as the NOD SCID mouse, lack functional adaptive immunity and can be further engineered to express or lack specific complement components. These models are particularly valuable for investigating the interplay between complement activity and immune cell function, as well as for human cell or tissue engraftment studies. They enable the assessment of humanized therapies and the study of complement-mediated pathology in the absence of confounding immune responses. Advantages include enhanced engraftment efficiency and the ability to model human disease mechanisms in vivo.

Transgenic and Knockout Combination Models

By combining transgenic overexpression and targeted gene knockout strategies, Protheragen develops complex models that replicate multifactorial aspects of complement-mediated pathology. For instance, C7 knockout/transgenic mice allow for the investigation of both loss- and gain-of-function effects in the same genetic background. These models are particularly useful for preclinical efficacy studies, biomarker discovery, and mechanistic research into gene-gene or gene-environment interactions. The main advantages are the flexibility to address diverse research questions and the ability to model both disease onset and progression.

Protheragen delivers a complete, end-to-end solution for in vivo Complement-Mediated Disease model development and testing. Our services encompass model generation, colony management, experimental design, therapeutic administration, and comprehensive phenotypic and molecular analysis. Key efficacy endpoints include survival, clinical scoring, complement activity assays (e.g., CH50, AH50), histopathology, cytokine profiling, and biomarker quantification. Analytical capabilities extend to flow cytometry, ELISA, immunohistochemistry, qPCR, and next-generation sequencing. Stringent quality control measures are implemented at every stage, including genetic validation, health monitoring, and standardized protocols to ensure data integrity and reproducibility.

Partnering with Protheragen provides access to cutting-edge animal models, scientific expertise, and a collaborative approach designed to accelerate your complement research and therapeutic development. Our commitment to scientific rigor, transparency, and client-focused solutions ensures that your research objectives are met efficiently and effectively. Contact us today to discuss your project needs and discover how our in vivo model services can advance your Complement-Mediated Disease research.