Gene Therapy Model Pack

Applied BioMath Assess™ Gene Therapy Model Pack includes 8 models covering LNP-encapsulated mRNA and siRNA therapies.

  • LNP-encapsulated mRNA expressing an intracellular protein in vitro
    • Two-compartment model of mRNA uptake, protein expression, and protein-mediated enzymatic reaction in vitro
  • LNP-encapsulated mRNA expressing an intracellular protein in vivo
    • Two-compartment model of mRNA delivery, protein expression, and protein-mediated enzymatic reaction in vivo
  • LNP-encapsulated mRNA expressing an extracellular protein in vitro
    • Two-compartment model of mRNA delivery, protein expression, and protein-mediated enzymatic reaction in vitro
  • LNP-encapsulated mRNA expressing an extracellular protein in vivo
    • Two-compartment model of mRNA delivery, protein expression, and protein-mediated enzymatic reaction in vivo
  • LNP- encapsulated siRNA expressing an intracellular protein in vitro
  • LNP-encapsulated siRNA expressing an intracellular protein in vivo 
  • LNP-encapsulated siRNA expressing an extracellular protein in vitro 
  • LNP-encapsulated siRNA expressing an extracellular protein in vivo

 

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Model Resources

Each model in the Gene Therapy Model Pack includes a pharmacology diagram (Figure 1) with model parameters and their nominal values (Figure 2).

 

Figure 1: Model Diagram

model I_mRNA

Created with BioRender.com

Figure 2: Model Parameters

Each model in Applied BioMath AssessGene Therapy Model Pack has a unique set of parameters, a sample of which is shown here, based on the model pharmacology and number of compartments. More details on the full list of parameters are available upon request. In the app, each parameter contains a default value based on expansive literature searches and references.

gene therapy model parameters

 

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