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Combining nonsense mutation suppression therapy with nonsense-mediated decay inhibition in neurofibromatosis type 1
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...
Autores principales: | , , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2023
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10384610/ https://www.ncbi.nlm.nih.gov/pubmed/37520682 http://dx.doi.org/10.1016/j.omtn.2023.06.018 |