Lesson 1Selecting in vitro models: organoids, co-culture systems, 2D vs 3D scaffolds and disease-relevant readoutsThis part talks pick and plan of lab models, with flat grows, 3D frames, organ bits, and joint grows, stressing surround hints, sickness-match checks, speed, and match with body finds.
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 spots on small critter models, mostly mouse and rat, describing sickness models for regrow cases, gene tools, guard backs, easy pluses, and main limits for guessing people safety and work.
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 shows dose plans for cell healings, with dose-range setups, one vs repeat gives, path-based tweaks, spread maps, hold and join checks, and model show-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 checks big critter models like pig, sheep, and ape-like, focusing body and work match, guard set, home and care, size of tools and doses, and when big ones key for move.
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 details pick and check end measures, with tissue looks, work tests, pic ways, molecule signs, and act checks, stressing feel, true, count, and tie to people clinic 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 looks at give paths and tool-help ways for cell and gene-tweak goods, with vein, in-organ, in-joint, frame-based, and tube ways, pointing aim good, safety, and tech do.
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 brings strict early test plan, from set guesses and pick holds to random, blind, size count, and number ends, aiming cut lean and better repeat and rule take.
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 covers get and make of early cell lots, with tissue pick, get out, grow big, freeze hold, quality watch, GMP follow, and let-go rules for steady, safe, well-known goods.
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
