How did you find a new the treatment for epidermolysis bullosa?
Dr. Tolar: We genetically engineered mice to have the same disease and then we planned all the possible strategies that could be helpful. We started from the source of the cells taken from the bone marrow. The reason for that is at least two-fold. The first is we have a 50 year history of bone marrow transplantation to show we can actually transfer cells from one individual to another and recreate the whole lymphoid system and blood forming system in the body. So we know how to do it. The second reason is that all the cells from the donor should be able to make collagen 7. The trick is, we didnt know which ones, and we didnt know whether the cells, even if they had the right gene for this protein, would actually express it. How did the bone marrow transplant work in the mice? Dr. Tolar: We took different types of cells from the bone marrow and we tested them one by one. There were about 16 of them and 15 didnt work. The one that we identified actually worked in about one quarter of the
