Application of gene therapies to regenerative medicine
& inherited skin disorders
Genome engineering is being applied to help advance regenerative therapies, including those using induced pluripotent stem cells.
We have undertaken experimental trials of lentiviral engineered skin cells for inherited skin diseases, Netherton Syndrome and Epidermolysis Bullosa. The results have been published and helped establish safety profiles for these new approaches. Further experimental lab work is underway to test emerging CRISPR strategies.