Lesson 1Selecting in vitro models: organoids, co-culture systems, 2D vs 3D scaffolds and disease-relevant readoutsThis part discuss picking and designing in vitro models, like 2D cultures, 3D scaffolds, organoids, and co-cultures, stressing surroundings cues, disease readouts, speed, and match with animal findings.
2D monolayer versus 3D culture systemsOrganoid models for organ-specific diseasesCo-culture and immune–stromal interactionsBiomaterial scaffolds and matrix mechanicsAssay readouts linked to in vivo outcomesThroughput, scalability, and assay robustnessLesson 2Small animal models: mouse and rat disease models relevant to chosen indication and translatability limitsThis part focus on small animal models, mostly mouse and rat, describing disease models for regenerative cases, genetic tools, immune setups, practical pluses, and main limits for predicting human safety and efficacy.
Mouse versus rat: strengths and weaknessesImmunodeficient and humanized rodent modelsCommon disease models for key indicationsModel validation and relevance to humansLimitations in scale, immunity, and lifespanBridging data to large animals and clinicsLesson 3Dosing strategies and kinetics: cell dose-ranging studies, biodistribution, and persistence analysisThis part explain dosing plans for cell therapies, including dose-range designs, single vs repeat dosing, route-based adjustments, biodistribution maps, persistence and engraftment checks, and modeling exposure-response links.
Design of dose-escalation and range-findingSingle versus multiple dosing regimensLabeling cells for tracking in vivoBiodistribution assessment and organ tropismPersistence, engraftment, and clearance kineticsExposure–response and safety margin modelingLesson 4Large animal models: pig, sheep, non-human primate choices for scale-up and functional similarityThis part examine large animal models like pig, sheep, and non-human primates, focusing on body and function similarity, immune context, housing and care, scaling devices and doses, and when big models are key for translation.
Criteria for choosing large animal speciesPig models for cardiovascular and liver repairSheep models for orthopedic and spine studiesNon-human primate models and ethicsScaling of devices, doses, and proceduresHusbandry, welfare, and regulatory oversightLesson 5Outcome measures: histology, functional assays, imaging modalities (MRI, PET, bioluminescence), molecular biomarkers, and behavioral testsThis part detail how to pick and validate outcome measures, like histology, function assays, imaging ways, molecular biomarkers, and behavior tests, stressing sensitivity, specificity, counting, and link to human clinical gain.
Histological scoring and morphometric analysisIn vitro and ex vivo functional assaysMRI, PET, and optical imaging strategiesMolecular biomarkers and omics readoutsBehavioral tests and functional performanceValidation, standardization, and assay QCLesson 6Delivery routes and device-assisted delivery: intravenous, intra-organ, intra-articular, biomaterial scaffolds, and catheter-based techniquesThis part review delivery routes and device help for cell and gene products, including IV, intra-organ, intra-joint, scaffold-based, and catheter ways, highlighting target efficiency, safety, and tech feasibility.
Intravenous and intra-arterial deliveryDirect intra-organ and parenchymal injectionIntra-articular and intradiscal approachesBiomaterial scaffolds and hydrogelsCatheter-based and image-guided deliveryProcedure-related risks and mitigationLesson 7Study design principles: hypotheses, controls, randomization, blinding, sample size calculation, and statistical endpointsThis part introduce strict preclinical study design, from setting hypotheses and controls to randomization, blinding, sample size calc, and stats endpoints, aiming to cut bias and boost reproducibility and regulator okay.
Formulating testable mechanistic hypothesesChoice of control and comparator groupsRandomization schemes and allocation concealmentBlinding of investigators and outcome assessorsSample size and power calculationsPrimary, secondary, and exploratory endpointsLesson 8Cell sourcing, manufacturing and GMP considerations for preclinical batches: isolation, expansion, cryopreservation, and release criteriaThis part cover sourcing and making preclinical cell batches, including tissue pick, isolation, expansion, freezing, quality control, GMP rules, and release criteria to ensure steady, safe, and well-known products.
Donor selection and tissue procurementCell isolation and early characterizationExpansion, passaging, and senescence controlCryopreservation methods and thaw recoveryGMP documentation and batch recordsRelease testing and specification setting
