A prospective phase I study of lentiviral-mediated COL7A1 gene-corrected autologous fibroblast therapy in adults with recessive dystrophic epidermolysis bullosa (LENTICOL-F)Completed
|Project lead||Prof John McGrath|
|Organisation||King’s College London, London, UK|
|Project budget||GBP 499,320.00|
|Start date / Duration||01. Feb 2015 / 43 months|
|Funder(s) / Co-Funder(s)||DEBRA UK, Others, MSAP/EBEP Recommended|
|Other funder(s)||Sohana Research Fund now CURE EB|
|Research area||Molecular therapy, Cellular therapy|
Short lay summary
Recessive dystrophic epidermolysis bullosa (RDEB) is caused by damage in a gene called COL7A1 that controls the production of a protein – type 7 collagen (C7) in the skin. C7 is an important sticky protein that glues the outer and inner layers of the skin together through hook-like structures called anchoring fibrils. In RDEB, the lack of C7 leads to blisters. Being able to restore C7 in the RDEB skin by gene therapy would lead to fewer blisters and stronger skin.
The team at St John’s Institute developed a gene therapy approach to try to restore C7 in RDEB skin. Laboratory research was carried out to make an artificial copy of the COL7A1 gene and to deliver the new gene into skin cells called fibroblasts. The fibroblasts are cells which are normally found in the inner layer of the skin. They can make collagens and other proteins that keep skin healthy.
Once the researchers had established that the skin cells could be corrected to make new C7 proteins, they carried out the essential safety checks and work to produce a high-grade gene therapy product that could be used in people with RDEB.
After this work was completed, the LENTICOL-F clinical trial was set up to test whether this form of gene therapy was safe to use in humans.