Combining nonsense mutation suppression therapy with nonsense-mediated decay inhibition in neurofibromatosis type 1

• Main Authors: Sara H. Osum (Author), Eunice I. Oribamise (Author), Stanislas M.A.S. Corbière (Author), Mandy Taisto (Author), Tyler Jubenville (Author), Alex Coutts (Author), Mark N. Kirstein (Author), James Fisher (Author), Christopher Moertel (Author), Ming Du (Author), David Bedwell (Author), David A. Largaespada (Author), Adrienne L. Watson (Author)
• Format: Book
• Published: Elsevier, 2023-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Neurofibromatosis type 1 (NF1) results from germline mutations in the tumor-suppressor gene NF1 and predisposes patients to developing nervous system tumors. Twenty percent of NF1 patients harbor nonsense mutations resulting in premature termination codons (PTCs). Nonsense suppression therapies can...