Nature paper reports epidermis regeneration on a strikingly large-scale.
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The patient has a mutation in gene called LAMB3 that causes junctional epidermolysis bullosa (JEB) so the core idea here was to replace the lost epidermis with new epidermis cells with wild type LAMB3 that would hopefully restore the patient’s healthy skin. The patient was in a dire situation after acutely losing 60% of his epidermis and having an overall ~80% loss. The team took a skin biopsy from a remaining relatively healthy area and used it to establish cell cultures, which were then transduced with a Moloney leukemia virus encoding a normal LAMB3 gene. They didn’t replace the mutant gene, but rather added a normal copy into the cells too to try to get some of the normal function back.
Transgenic epidermal sheets expressing the wild type gene were grafted onto the patient and remarkably the patient has had a near complete restoration of his epidermis as a result....
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