AAV-mediated brain antibody delivery and TargetX binding
A reduced QSP model linking AAV biodistribution and nuclear persistence to transgene expression, antibody production in plasma, brain interstitial fluid, and CSF, and reversible TargetX binding.
- Therapeutic
- AAV-encoded anti-TargetX antibody, anti-TargetX mAb
- Target
- TargetX (anonymized paper target)
- Disease
- Rat brain AAV gene delivery and target engagement
Biological model
A reduced QSP model linking AAV biodistribution and nuclear persistence to transgene expression, antibody production in plasma, brain interstitial fluid, and CSF, and reversible TargetX binding.
Model details
A reduced QSP model linking AAV biodistribution and nuclear persistence to transgene expression, antibody production in plasma, brain interstitial fluid, and CSF, and reversible TargetX binding.
Modeled states
- AAV fast csf1
Modeled dynamic state for aav fast csf.
- AAV fast isf1
Modeled dynamic state for aav fast isf.
- AAV fast plasma1
Modeled dynamic state for aav fast plasma.
- AAV nuclear csf1
Modeled dynamic state for aav nuclear csf.
- AAV nuclear isf1
Modeled dynamic state for aav nuclear isf.
- AAV nuclear plasma1
Modeled dynamic state for aav nuclear plasma.
- complex csfnM
Modeled dynamic state for complex csf.
- complex isfnM
Modeled dynamic state for complex isf.
- complex plasmanM
Modeled dynamic state for complex plasma.
- mAb csfnM
Modeled dynamic state for mab csf.
- mAb isfnM
Modeled dynamic state for mab isf.
- mAb plasmanM
Modeled dynamic state for mab plasma.
Key readouts
- Plasma anti-TargetX antibodyunknown
Model-derived readout for plasma anti-targetx antibody.
- Brain-ISF anti-TargetX antibodyunknown
Model-derived readout for brain-isf anti-targetx antibody.
- CSF anti-TargetX antibodyunknown
Model-derived readout for csf anti-targetx antibody.
- Free TargetX in brain ISFunknown
Model-derived readout for free targetx in brain isf.
- Antibody–TargetX complex in brain ISFunknown
Model-derived readout for antibody–targetx complex in brain isf.
Explore this model
Choose a starting point to view its result. Adjust key model inputs when you want to explore a different outcome.
IST 2.5E11 Vg AAV9
How do AAV route and dose propagate through transgene expression to antibody exposure and TargetX engagement in plasma, brain ISF, and CSF? Explore this biological starting configuration through Plasma anti-TargetX antibody, Brain-ISF anti-TargetX antibody, CSF anti-TargetX antibody, Free TargetX in brain ISF, Antibody–TargetX complex in brain ISF. This is a mechanistic product exploration, not a paper-result reproduction.
Questions to explore
- How do AAV route and dose propagate through transgene expression to antibody exposure and TargetX engagement in plasma, brain ISF, and CSF?
Starting result
This result reflects the starting settings. Run your changes to update it.
Model inputs
Five scientist-facing controls at most.
Compare a parameter
How does one model input change the response?
How do AAV route and dose propagate through transgene expression to antibody exposure and TargetX engagement in plasma, brain ISF, and CSF?
k aav fast loss h
Model parameter controlling k aav fast loss h.
Exploratory range around the default value.
5 evenly spaced values in 1/h. Other model inputs and the simulation window stay fixed.
Interpret with care
- Interpret trajectories as deterministic model behavior, not as a patient-specific prediction or dosing recommendation.
- Paper-result and exact-anchor checks remain in the private validation lane and are not part of this public scenario.
Review the server-confirmed fixed price before starting the comparison.
Comparative response
End-of-window response across the selected parameter values.
Choose an exploratory range around the default, review the fixed price, and run the comparison.
Scientific reference
Supporting publication
QSP model for AAV-mediated antibody delivery in rat brainJournal of Pharmacokinetics and Pharmacodynamics