However, all present were fully conscious of the many hurdles still to be overcome in this three year programme of work. In particular, it was felt to be of overwhelming importance that people living with EB had a clear understanding of the exploratory nature of the work and that even successful completion of the full programme of work would not mean that treatments would be immediately available.

Skintherapy is concerned with both dominant and recessive dystrophic EB and it was noted that, whilst severe forms of RDEB where there is an absence of collagen VII could serve as a good test for the effectiveness of vectors, there was a high risk that rejection might take place since the body would not recognise the introduced collagen VII. It is probable that milder forms of RDEB and DDEB, where mutated collagen VII is present, would be better candidates, at least initially, for gene therapy.

Much discussion focused on the choice of vector. Essentially the choice is between retrovirals, which are available now, lentivirals, which should come on line over the next few years and hybrid AAV/adenoviral vectors which, although potentially the most effective, are still a long way off. The debate was whether to move forward with what would be available and, thereby, make Phase I clinical trials (to assess toxicity and safety but not effectiveness) possible as a follow on to the Skintherapy programme of work or to wait for a better vector which might never come. Safety assessments that had been carried out suggested to the scientists present that the balance of advantage lay with pressing on with what we expect to have, but it was recognised that this is a choice in which patients should have a strong input.

Encouragement was taken from the news reported by Prof. Michele de Luca that a Phase I clinical trial had re-commenced on 6 October 2005 with a single patient with non-Herlitz JEB (the same patient who had participated in the original, aborted 2002 trial). However, it was noted that in Italy the moratorium on gene therapy trials involving retroviruses remains in force and that trials for DEB would face significant obstacles in gaining regulatory approval in Europe.