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Suppression of NF1 Nonsense Mutations by RNA-guided RNA Pseudouridylation

A significant proportion (≈20%) of NF1 mutations generate a premature “stop” signal in the middle of the NF1 gene, leading to a truncated and non-functional …
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Development of Mutation Suppression Therapy in a Swine Model of NF1

One in five neurofibromatosis type 1 (NF1) patients have nonsense mutations in the NF1 gene that causes its protein production machinery to stop early, resulting …
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Ataluren-promoted Therapeutic Nonsense Suppression for Neurofibromatosis

Nonsense codons are words in the genetic code that instruct the gene expression apparatus to stop the process of protein synthesis at a precise point, …
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Exploring Nonsense Suppression as a Treatment for NF1

Nonsense mutations are gene lesions that introduce a premature termination codon (PTC) into a messenger RNA (mRNA). Since PTCs are found in a significant number …
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