GeneticsMolecular Therapy — Nucleic AcidsPeer-reviewed
NF2 lacking exon 11 isoform induced by antisense gene therapy is able to recover partially merlin deficiency on NF2-related Schwannomatosis iPSC-derived spheroid model
Gemma Casals-Sendra, Ignasi Jarne-Sanz, Núria Catasús…·Elisabeth Castellanos
This study shows that antisense gene therapy using PMOs can induce exon 11 skipping in NF2-deficient Schwann cells, partially restoring merlin function and key pathways in a spheroid model of NF2-SWN.
DOI 10.1016/j.omtn.2026.103046