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Article

Nonsense-mediated mRNA decay and cystic fibrosis.

Methods in molecular biology (Clifton, N.J.) - 1 Jan 2011

Linde Liat, Kerem Batsheva

Abstract excerpt

Approximately one-third of the alleles causing genetic diseases carry premature termination codons (PTCs). Therapeutic approaches for mutations generating in-frame PTCs are aimed at promoting translational readthrough of the PTC, to enable the synthesis and expression of full-length functional proteins. Interestingly, readthrough studies in tissue culture cells, mouse models, and clinical trials revealed a wide...

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Identifiers

PMID: 21594783
DOI: 10.1007/978-1-61779-117-8_10

Genes

CFTR

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