Preclinical translation of Neurofibromatosis type 1 (NF1) exon 17 skipping using targeted U7-SnRNA packaged into engineered AAV serotypes.
Marc Moore, Kimia Rayat-Sanati, Xiaoxia Zhang, Hui Liu, Zakaria Rostamitehrani, Tarah Vijayasarathy, Erik Westin, Miguel Sena‐Esteves, Casey A. Maguire, Robert A. Kesterson, Linda J. Popplewell, DEEANN E. WALLIS
This paper evaluates AAV serotypes for delivering U7-SnRNA to induce NF1 exon 17 skipping in mice, finding AAV-F is most effective in brain and AAV-B1 in sciatic nerve.
bioRxiv · Jul 2, 2026 · PreprintGenetics