Putting a stop to nonsense: Revisiting gene correction therapy for neurofibromatosis type 1
Author:
Funder
National Institute of Neurological Disorders and Stroke
Publisher
Elsevier BV
Subject
Drug Discovery,Molecular Medicine
Reference10 articles.
1. Combining nonsense mutation suppression therapy with nonsense mediated decay inhibition in Neurofibromatosis type 1;Osum;Mol. Ther. Nucleic Acids,2023
2. Inhibition of nonsense-mediated mRNA decay may improve stop codon read-through therapy for Duchenne muscular dystrophy;Amar-Schwartz;Hum. Mol. Genet.,2023
3. VX-445-Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles;Keating;N. Engl. J. Med.,2018
4. Biochemical and structural analyses reveal that the tumor suppressor neurofibromin (NF1) forms a high-affinity dimer;Sherekar;J. Biol. Chem.,2020
5. Mechanism for the learning deficits in a mouse model of neurofibromatosis type 1;Costa;Nature,2002
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1. Therapeutic Nonsense Suppression Modalities: From Small Molecules to Nucleic Acid-Based Approaches;Biomedicines;2024-06-10
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