Figure 2.

Endogenous trans-splicing: Gene and protein expression in RDEB-KC. (A) Schematic of the endogenous trans-splicing process. The complete transcript region from exon 1 to 64, which harbors the mutation of interest, is replaced by its wild-type copy provided by RTMe. (B) SqRT-PCR performed on an RDEB patient cell line retrovirally transduced with RTMeA and RTMeB. Means ± SD of fold changes of COL7A1 expression over wild-type keratinocytes (KC) of three independent experiments are given. Unpaired, two-sided Student’s t-test was performed and p-values ≤ 0.05 were considered significant (*). (C) Western blot analysis showed re-expression and secretion of C7.