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, Irene Boluda-Luis, Ariadna Quer, Emilio Amilibia, Scott Randall Plotkin, Conxi Lázaro, Eduard Serra, Ignacio Blanco, 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.
Molecular Therapy — Nucleic Acids · Aug 3, 2026Peer-reviewedGenetics