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. 2019 Aug 8;28(10):1176–1189. doi: 10.1111/exd.13979

Table 1.

Facts from JEB gene replacement therapies

Genetically corrected epidermal grafts LAMB3
  • Showed regeneration of morphologically normal, fully differentiated and functional, mechanically stable and non‐blistering transgenic skin
  • Can replace up to 80% of a patient's diseased skin84
  • Showed sustained synthesis of the transgenic protein131
  • Harbour EpSCs which expanded and ensured long‐term or permanent regeneration84
  • Despite the risk of insertional mutagenesis tumorigenicity has not been observed within a follow‐up period of currently more than 12 y (83, 84, 131, and unpublished observations)
  • Following regeneration, there was no evidence of an immune response against the newly expressed protein (unpublished observations)