Genetic Kidney Disease Models
At Ace Therapeutics, we specialize in preclinical research services for genetic kidney disorders, offering tailored solutions to support drug development, target validation, and mechanistic studies. Our validated genetic models replicate human disease pathology, enabling robust preclinical data generation with translational relevance.
Genetic kidney diseases, driven by inherited mutations, require models that faithfully mimic human molecular pathology. These models are essential for
Our genetic kidney disease platform supports end-to-end preclinical studies, including
Dose-response studies for small molecules, antisense oligonucleotides (ASOs), or gene therapies Biomarker correlation with histological and functional outcomes
Pathway analysis (e.g., mTOR signaling in PKD, autophagy in FSGS) Single-cell RNA sequencing to map cell-type-specific responses
Urinary exosome analysis for podocyte-derived markers Proteomic profiling of disease progression biomarkers
MRI-based cyst volumetry for PKD models Confocal microscopy for glomerular injury quantification
Our platform integrates established and custom models to address diverse research needs. Below are key models and their validation criteria
| Disease Model | Species/Strain | Key Validation Endpoints |
| Alport Syndrome | Col4a3/4/5 KO mice | Proteinuria, GBM ultrastructure (EM), collagen IV staining |
| FSGS | Nphs1/Nphs2 KO mice | Podocyte effacement, albuminuria, histopathology |
| Fabry Disease | Gla KO mice | Globotriaosylceramide (Gb3) accumulation, renal fibrosis |
| Polycystic Kidney Disease | Pkd1 conditional KO rats | Cyst volume (MRI), renal function decline, fibrosis |
| C3 Glomerulopathy | Cfh-/- mice | C3 deposition (IF), complement activation markers |
Every model undergoes rigorous quality control:
Our models are optimized for
What distinguishes genetic models from induced injury models?
Genetic models replicate inherited molecular defects driving chronic kidney disease, enabling studies of disease initiation and progression, unlike acute injury models.
Can you develop custom knockouts for novel targets?
Yes. Our CRISPR/Cas9 platform supports generation of conditional or tissue-specific KO models.
How do you validate model relevance to human disease?
We correlate molecular markers (e.g., collagen IV variants in Alport mice) with human biopsy data and clinical progression patterns.
What endpoints are measured in Fabry disease studies?
Standard endpoints include Gb3 accumulation (LC-MS/MS), pain response assays, and cardiac/renal fibrosis markers.
Do you offer biodistribution studies for gene therapies?
Yes. We perform qPCR-based vector tracking and tissue-specific expression analysis in target organs.
Make Order
Experimental Scheme
Implementation
Conclusion
Ace Therapeutics has a group of highly committed and skilled researchers with the objective to provide innovative preclinical contract research solutions to cope with kidney diseases.
We strives to accelerate the development of innovative therapies that address unmet medical needs.