TY  - RPRT
TI  - Preclinical translation of Neurofibromatosis type 1 (NF1) exon 17 skipping using targeted U7-SnRNA packaged into engineered AAV serotypes.
AU  - Moore, M.
AU  - Rayat-Sanati, K.
AU  - Zhang, X.
AU  - Liu, H.
AU  - Rostamitehrani, Z.
AU  - Vijayasarathy, T.
AU  - Westin, E.
AU  - Esteves, M.
AU  - Maguire, C. A.
AU  - Kesterson, R. A.
AU  - Popplewell, L.
AU  - Wallis, D.
PY  - 2026
DO  - 10.64898/2026.06.29.734312
UR  - https://www.biorxiv.org/content/10.64898/2026.06.29.734312v1
ID  - 10.64898/2026.06.29.734312
ER  -