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Holo-GT focuses on developing innovative gene therapy techniques for dominant genetic skin diseases, using Epidermolysis Bullosa as a model. The project aims to create a safe protocol for gene editing in skin cells to address the challenges posed by dominant forms of the disease.
Holo-GT will define innovative ex vivo gene therapy of dominant genetic skin disease, by using Epidermolysis Bullosa (EB) as a model system. EB is a group of dominantly or recessively inherited, devastating, incurable diseases marked by structural fragility of the integuments.
The applicant has shown that combined ex vivo cell and gene therapy can cure the skin of recessive LAMB3-Junctional EB.
This lifesaving procedure unveiled that the human epidermis is sustained solely by long-lived stem cel…
UNIVERSITA DEGLI STUDI DI MODENA E REGGIO EMILIA
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