NF2 lacking exon 11 induced by antisense gene therapy is able to partially recover merlin deficiency in NF2-SWN iPSC-derived spheroid model
Results confirm that the PMO treatment induces effective skipping of exon 11 in Schwann cell spheroids, generating a hypomorphic merlin_e-11 that has the capacity to partially rescue merlin-deficiency in an NF2-SWN spheroid cell model and that it is a potential treatment of patients who harbor truncating variants locat...