How gene therapy was used to treat a skin disease

Patient had lost 80% of his skin to a blistering disease

Epidermal cells were taken from a non-blistering area of skin ...

Blistered

skin

Epidermal cells

Colonies of cells with corrected genes were then grown in the lab

... and genetically modified using a virus to correct a defect in a gene called LAMB3

Retrovirus

LAMB3

The cell colonies grew into sheets of genetically modified skin – enough to cover almost the entire body of the patient

Over several months, a patchwork of grafts became completely integrated and started to self-renew, just as ordinary skin does

Patient had lost 80% of his skin to a blistering disease

Colonies of cells with corrected genes were then grown in the lab

Epidermal cells were taken from a non-blistering area of skin ...

... and genetically modified using a virus to correct a defect in a gene called LAMB3

Infection

Blistered

skin

Epidermal cells

Retrovirus

LAMB3

The cell colonies grew into sheets of genetically modified skin – enough to cover almost the entire body of the patient

Over several months, a patchwork of grafts became completely integrated and started to self-renew, just as ordinary skin does