In an ex-vivo CRISPR therapy, cells are extracted from the patient, edited outside the body with a Cas9/guide-RNA complex delivered by a viral vector, electroporation, or lipid nanoparticle (LNP), then reinfused. Each delivery method has a characteristic efficacy factor; higher editing dose raises the on-target edit rate but also raises off-target risk along a dose-response curve.
Edit success = base_rate(delivery) × dose_response(dose)
Off-target rate ∝ dose² × (1 − delivery_fidelity)
Clinical response ≈ f(successful edit rate, cohort variability)
- Editing dose — Cas9/gRNA concentration applied per cell; raises edit efficiency but increases off-target cutting risk.
- Delivery method — viral vector (AAV, high efficiency, immunogenic), electroporation (moderate, physical), or LNP (gentler, lower penetration) — each rescales success/off-target curves.
- Cohort size — number of simulated patients whose cells move through the pipeline, feeding the live outcome bar chart.
- Run pipeline / Step patient — animate the extraction→edit→reinfusion cycle continuously or advance one patient at a time.
This mirrors real ex-vivo trials such as CRISPR-based sickle-cell and beta-thalassemia therapies, where hematopoietic stem cells are edited outside the body before reinfusion.