Lesson 1Selecting in vitro models: organoids, co-culture systems, 2D vs 3D scaffolds and disease-relevant readoutsThis section discusses picking and setting up lab models, like flat cultures, 3D supports, mini-organs, and mixed cell systems, stressing surrounding signals, illness-linked checks, speed, and match with live body results.
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 section centres on small creatures like mice and rats, describing illness setups for repair needs, gene tools, immune setups, hands-on benefits, and main limits in foretelling human safety and gains.
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 section explains dosing plans for cell treatments, including range tests, one-time vs repeated, path-based tweaks, spread mapping, lasting presence checks, and modelling dose-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 section looks at big animals like pigs, sheep, and apes, focusing on body and function likeness, immune setting, care needs, scaling tools and doses, and when big models are key for human shift.
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 section details choosing and checking result measures, like tissue slices, function tests, scan types, molecule signs, and behaviour checks, stressing accuracy, detail, counting, and link to human health gains.
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 section reviews paths and tool-aided ways for cell and gene-altered items, including vein, organ-inside, joint, support-based, and tube methods, highlighting aim accuracy, safety, and doability.
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 section introduces solid early study planning, from setting questions and controls to random grouping, hiding info, size figuring, and stat goals, to cut bias and boost repeat and approval chances.
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 section covers getting and making early cell lots, including tissue pick, pulling out, growing, freezing, quality watch, good practice rules, and release standards for steady, safe, 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
