Gene Editing Simulator

A single base change makes hemoglobin sticky. Red blood cells become rigid and sickle-shaped, blocking blood vessels and causing pain crises.

BCL11A silences fetal hemoglobin. Disrupting its enhancer reactivates fetal hemoglobin to compensate for defective adult hemoglobin.

Overactive PCSK9 destroys LDL receptors on liver cells, preventing cholesterol clearance. LDL skyrockets, causing early heart attacks.

Mutant TTR protein misfolds and deposits as amyloid fibrils in nerves and heart, causing progressive neuropathy and cardiomyopathy.

A frameshift mutation stops dystrophin production. Without this structural protein, muscle fibers break down progressively with every contraction.

Expanded CAG repeats (>36) produce a toxic huntingtin protein with a long polyglutamine tract that forms aggregates and kills neurons.

Deletion of phenylalanine at position 508 causes CFTR protein to misfold. Defective chloride channels lead to thick, sticky mucus in lungs.

HIV uses the CCR5 receptor to enter T-cells. Knocking out CCR5 blocks viral entry, mimicking the natural CCR5-Δ32 mutation found in ~1% of Europeans.