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Translational Disease Modeling
SignaGen bridges in vitro discovery and in vivo modeling by providing tissue-specific AAV serotypes and pathogenic gene delivery solutions.
AAV vectors are widely used for disease modeling because of their strong safety profile, low immunogenicity, and ability to support long-term gene expression in both dividing and non-dividing cells. Compared with conventional transgenic approaches, AAV-based models enable faster development and precise spatial and temporal control of gene expression through tissue-specific promoters and distinct serotypes. For example, targeted intracranial delivery of AAV can be used to model neurodegenerative disorders such as Alzheimer’s and Parkinson’s disease.
SignaGen offers a comprehensive portfolio of off-the-shelf and custom AAV vectors for efficient generation of animal disease models, including Alzheimer’s disease, Parkinson’s disease, Huntington’s disease, hepatitis, and diabetes.
Off-the-shelf AAV vectors for translational disease modeling
AAV vectors are widely used to generate in vivo animal models by delivering disease-relevant genes (or gene-silencing/editing components) directly to target tissues, enabling controlled, reproducible pathology without the time and variability of traditional breeding.
Alzheimer’s disease models: AAV enables region-specific expression of AD drivers such as mutant APP, PSEN1/PSEN2, or tau (MAPT), often in hippocampus and cortex, inducing key features like amyloid pathology and/or tauopathy, synaptic dysfunction, and cognitive deficits. These models are useful for assessing pathway biology, biomarkers, and therapeutic modalities (antibodies, gene therapy, and combination approaches) in a controlled, scalable way.
Hepatitis B (HBV) models: AAV can deliver HBV genomes or HBV replicons to hepatocytes (typically via liver-tropic serotypes), producing sustained hepatic expression of viral antigens and replication intermediates. These models are commonly used to study chronic HBV antigenemia, host immune responses, and to evaluate antivirals, neutralizing antibodies, and gene-silencing/editing approaches aimed at reducing HBV markers.
Huntington’s disease models: AAV can be injected into the striatum (or connected circuits) to express mutant huntingtin (mHTT) fragments or full-length constructs with expanded CAG repeats. This produces robust, localized neurodegeneration and motor/behavioral phenotypes, supporting rapid testing of allele-selective silencing, miRNA/shRNA, CRISPR-based strategies, and neuroprotective therapeutics.
Parkinson’s disease models: Overexpression of the human SNCA (hSNCA) leads to elevated levels of α-synuclein, a presynaptic protein that readily misfolds and aggregates. This accumulation promotes the formation of Lewy bodies, a hallmark of Parkinson’s disease, and disrupts synaptic function and neuronal homeostasis. As a result, dopaminergic neurons in the substantia nigra undergo progressive degeneration, driving disease onset and progression.
Explore our pre-made AAV vectors for establishing in vivo animal disese models.
| SKU # | Viral Vector | Unit Size | Titer |
|---|---|---|---|
| SL116257 | AAV8-HBV1.2 | 10 ~ 30 µL | >1E+13 VG/mL |
| SL116258 | AAV8-HBV1.3 | 10 ~ 30 µL | >1E+13 VG/mL |
| SL116503 | AAV8-HBV1.0 | 10 ~ 30 µL | >1E+13 VG/mL |
| SL116525 | AAV8-WMHBV | 10 ~ 30 µL | >1E+13 VG/mL |
| SL116515 | AAV2-CAG-GFP-httQ23 | 10 ~ 30 µL | >1E+13 VG/mL |
| SL116516 | AAV2-CAG-GFP-httQ72 | 10 ~ 30 µL | >1E+13 VG/mL |
| SL116517 | AAV(PHP.eB)-CAG-GFP-httQ23 | 10 ~ 30 µL | >1E+13 VG/mL |
| SL116518 | AAV(PHP.eB)-CAG-GFP-httQ72 | 10 ~ 30 µL | >1E+13 VG/mL |
| SL116519 | AAV2-Syn-GFP-httQ23 | 10 ~ 30 µL | >1E+13 VG/mL |
| SL116520 | AAV2-Syn-GFP-httQ72 | 10 ~ 30 µL | >1E+13 VG/mL |
| SL116521 | AAV(PHP.eB)-Syn-GFP-httQ23 | 10 ~ 30 µL | >1E+13 VG/mL |
| SL116522 | AAV(PHP.eB)-Syn-GFP-httQ72 | 10 ~ 30 µL | >1E+13 VG/mL |
| SL116523 | AAV(PHP.eB)-Syn-httQ18 | 10 ~ 30 µL | >1E+13 VG/mL |
| SL116524 | AAV(PHP.eB)-Syn-httQ150 | 10 ~ 30 µL | >1E+13 VG/mL |
| SL116526 | AAV9-hSyn-TAU(P301L) | 10 ~ 30 µL | >1E+13 VG/mL |
| SL116527 | AAV9-EF1a-EGFP-P2A-TAU(P301L) | 10 ~ 30 µL | >1E+13 VG/mL |
| SL116528 | AAV9-CAG-EGFP-P2A-TAU(P301L) | 10 ~ 30 µL | >1E+13 VG/mL |
| SL116529 | AAV9-CBh-TAU(P301L-S320F)-FLAG | 10 ~ 30 µL | >1E+13 VG/mL |
| SL116530 | AAV8-hSyn-TAU(P301L) | 10 ~ 30 µL | >1E+13 VG/mL |
| SL116531 | AAV9-CAG-Tau(P301L) | 10 ~ 30 µL | >1E+13 VG/mL |
| SL116532 | AAV9-CAG-Tau(P301S) | 10 ~ 30 µL | >1E+13 VG/mL |
| SL116533 | AAV9-CBh-TAU(P301L-S320F)-mCherry | 10 ~ 30 µL | >1E+13 VG/mL |
| SL116534 | AAV9-CBh-TAU(P301S)-mCherry | 10 ~ 30 µL | >1E+13 VG/mL |
| SL116535 | AAV9-CAG-Flex-GFP-Tau(P301L) | 10 ~ 30 µL | >1E+13 VG/mL |
| SL116536 | AAV(PHP.eB)-Syn-hSNCA | 10 ~ 30 µL | >1E+13 VG/mL |
| SL116537 | AAV(PHP.eB)-Syn-hSNCA-P2A-GFP | 10 ~ 30 µL | >1E+13 VG/mL |
| SL116539 | AAV9-CAG-mCherry-P2A-hSNCA | 10 ~ 30 µL | >1E+13 VG/mL |
| SL116540 | AAV9-Syn-mCherry-P2A-hSNCA | 10 ~ 30 µL | >1E+13 VG/mL |
Interested in custom AAV vectors for in vivo disease modeling?
SignaGen provides custom viral vector solutions for robust in vivo disease modeling. We design and produce AAV, lentiviral, and adenoviral vectors for gene overexpression, knockdown, and CRISPR-based editing. Our vectors are widely used to establish models of Huntington’s disease, Hepatitis B, and Alzheimer’s disease. With optimized design, high-titer production, and rigorous QC, we support reliable modeling across diverse animal systems.
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