22. Do you consider that the event will attract press interest?:
23. If yes, please summarise up to 4 key points you think will be of press interest:
Stem cells, gene therapy, 'molecular scissors' (designer nucleases such as TALE and zinc-finger nucleases), inherited blistering skin diseases.
24. Do you have any other comments you would like to make or information you would like to include?:
The Newcastle group is the only research group in the UK using 'molecular scissors' to develop a treatment for blistering skin diseases. Treatment of skin stem cells will ensure a permanent cure of the disease as this is the cell type which is responsible for skin regeneration. We have recently shown that 'molecular scissors' work in mouse skin stem cells (Höher et al, 2012, Stem Cell Reports and Reviews 8:426-34) which led to a press release by our funder, the British Skin Foundation, and subsequent press coverage. 'Molecular scissors' have great potential for various types of gene therapies. The first clinical trial using 'molecular scissors' aiming to prevent disease progression in HIV patients is currently ongoing in the USA. Newcastle University, with its North East Stem Cell Institute (NESCI) including a running 'Good Manufacturing Practice' (GMP) facility, and its close connection to the Royal Victoria Infirmary (RVI) is an ideal site for our research, for subsequent first in man trials, and, future applications of our therapy approach in patients.