Targeted exon skipping of NF1 exon 17 as a therapeutic for neurofibromatosis type I

• Main Authors: André Leier (Author), Marc Moore (Author), Hui Liu (Author), Michael Daniel (Author), Alexis M. Hyde (Author), Ludwine Messiaen (Author), Bruce R. Korf (Author), Jamuna Selvakumaran (Author), Lukasz Ciszewski (Author), Laura Lambert (Author), Jeremy Foote (Author), Margaret R. Wallace (Author), Robert A. Kesterson (Author), George Dickson (Author), Linda Popplewell (Author), Deeann Wallis (Author)
• Format: Book
• Published: Elsevier, 2022-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

We investigated the feasibility of utilizing an exon-skipping approach as a genotype-dependent therapeutic for neurofibromatosis type 1 (NF1) by determining which NF1 exons might be skipped while maintaining neurofibromin protein expression and GTPase activating protein (GAP)-related domain (GRD) fu...