Patient-related strategies to improve gene editing strategies for the treatment of EB

New scientific publication from the Koller Working Group

The aim of every EB gene therapy is to correct the gene mutation in order to ensure the production of a functional protein, and thus the cohesion of the skin layers. We have reported on several studies performed at the EB House, in which the modern gene editing methods TALEN and CRISPR were successfully applied in EB skin cells in cell culture. In contrast to previous gene therapies, these gene editing methods can permanently correct a mutation in the skin cells without the use of viruses, and therefore represent a safer and more efficient treatment option for EB. The CRISPR method in particular is developing rapidly and is constantly optimized, so that its application in an ex vivo gene therapy approach for EB is within reach.

Gene editing tools cut the double-stranded DNA at a specific target site in order to replace the mutated sequence with a correct one. Depending on the form of EB, the type of mutation and the nature of inheritance (dominant or recessive), different editing strategies have to be used, to obtain most effective results in individual cases. This is in line with observations from the EB House scientists that the efficiency of protein correction in EB skin cells through CRISPR or TALEN varies according to the gene editing strategy used.

In a review article recently published in the journal Cells, the EB House researchers describe five different gene editing strategies and their mode of action. The article discusses which strategy is best or less suitable for which EB patient, and points out the importance of the careful and individual choice of the gene editing strategy applied in a potential clinical application. Besides choosing the gene editing strategy in order to achieve the best therapeutic result, general safety aspects must be addressed before these technologies can be used in humans.

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