GeneticsbioRxivPreprint
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…·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.
DOI 10.64898/2026.06.29.734312