Disease Model Development Services
Protheragen advances rare liver disease research through specialized model development services spanning cellular, animal, and organoid systems. Our team leverages cutting-edge technologies to support mechanistic exploration and accelerate drug discovery, offering tailored solutions for both foundational studies and preclinical trials. Partner with us to advance your research objectives and translate insights into therapeutic strategies for rare liver conditions.
Overview of Rare Liver Disease Models
Rare liver diseases-encompassing metabolic disorders (e.g., Wilson disease, alpha-1 antitrypsin deficiency) and cholestatic conditions (e.g., PFIC, Alagille syndrome)-pose significant drug development challenges due to their intricate pathophysiology and small patient cohorts. Conventional preclinical models, including standard cell lines and animal systems, frequently lack the capacity to replicate human disease mechanisms, contributing to high failure rates in early-stage research. This underscores the necessity for advanced, clinically predictive models to bridge the gap between preclinical findings and therapeutic applications.
Key Advancements Include:
- Genetic Engineering: CRISPR/Cas9-edited animal models (e.g., ATP7B knock-in/knockout for Wilson disease) enabling precise replication of rare mutations.
- Humanized Systems: Liver-humanized mouse models (e.g., uPA) for evaluating human-specific drug metabolism and viral infection dynamics.
- Patient-Derived Models: 3D organoid cultures generated from patient biopsies, retaining disease-specific genetic and phenotypic characteristics.
- Integrated Platforms: Microphysiological systems (e.g., liver-on-a-chip) to simulate disease progression and assess drug responses under controlled conditions.
These innovations enhance mechanistic studies, high-throughput drug screening, and preclinical safety/efficacy evaluations, while improving regulatory alignment for IND-enabling research.
Fig1. Schematic diagram of in vitro liver organoid model creation. (Obeid, et al., 2024)Our Services
Protheragen delivers specialized disease modeling services for rare liver disorders, leveraging extensive expertise in rare disease research. We support drug discovery by developing, validating, and characterizing advanced models for drug screening and therapeutic efficacy evaluation. Our customizable disease models enhance preclinical research data quality, accelerating therapeutic development for rare hepatic diseases through biologically relevant experimental platforms.
Cell Model Development
We provide integrated model development services for liver disease research across three core platforms. In cell model development, we deliver customized solutions including cell line engineering for stable metabolic/drug screening platforms, primary hepatocyte isolation from human/animal sources for short-term assays, patient-specific iPSC-derived hepatocytes for genetic disease modeling, and physiologically relevant co-culture systems combining Kupffer cells, stellate cells, and endothelial cells to mimic liver microenvironment interactions.
Organoid Model Development
Our organoid development leverages 3D culture technologies to generate structurally/functionally competent liver models from adult stem cells (ASC), embryonic stem cells (ESC), and patient-derived samples (POD), with specialized tumor avatar services offering PDX models for Fibrolamellar HCC and rare hepatic cancers to support precision oncology research.
Animal Model Development
For animal models, we engineer genetic variants, humanized systems with transplanted liver/immune components, and chemically/physically induced disease phenotypes, complemented by customized behavioral testing protocols to strengthen preclinical data packages. All platforms are optimized to recapitulate rare liver disease pathologies with translational fidelity across cellular, tissue, and systemic levels.
Our models support:
- Target validation for gene therapy, RNAi, and small-molecule drugs
- Drug repurposing screens using organoid biobanks
- Toxicity testing of liver-targeted therapies (e.g., GalNAc-siRNA conjugates)
- Mechanistic studies of disease pathways (e.g., ER stress in AATD)
Why Choose Us?
- Expert Team
Seasoned scientists with deep expertise in rare liver disease research drive our projects. - Advanced Technologies
Precision gene editing, stem cell differentiation systems, and 3D bioprinting enable robust model development. - Customized Services
Client-specific models are engineered to match research objectives with biological relevance. - Quality Control
Multi-stage validation protocols confirm model reproducibility and clinical correlation. - Comprehensive Support
Integrated services cover model creation, compound screening, and preclinical validation workflows.
FAQs?
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Q: How long does it take to develop a custom rare liver disease model?
A: Timelines depend on complexity: basic cell models require 4-6 weeks, while complex models (e.g., PDX or animal models) take 12-16 weeks.
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Q: Can your models be used for research and preclinical testing?
A: Yes. Our models are validated for both mechanistic studies and preclinical drug screening, including efficacy and safety evaluations.
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Q: Why are Protheragen's models more reliable?
A: We employ CRISPR/Cas9, 3D bioprinting, and strict validation processes to ensure models accurately replicate disease biology and generate reproducible data.
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Q: Do you provide end-to-end support?
A: Yes. Our team provides comprehensive support from initial consultation through final delivery, including design, validation, and protocol optimization to meet your project goals.
Reference
- Obeid DA.; et al. Using Liver Organoids as Models to Study the Pathobiology of Rare Liver Diseases. Biomedicines. 2024;12(2):446.
All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.