Antisense oligonucleotides targeting exon 11 are able to partially rescue the Neurofibromatosis Type 2 phenotype in vitro
Núria Catasús, Inma Rosas, Sandra Bonache, Alejandro Negro, Miguel Torres‐Martín, Adrià Plana‐Pla, Héctor Salvador, Eduard Serra, Ignacio Blanco, Elisabeth Castellanos
This study shows that antisense oligonucleotides targeting exon 11 can partially rescue the NF2 phenotype in vitro by inducing a hypomorphic Merlin isoform.
bioRxiv · Feb 11, 2022 · PreprintGenetics