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Article

Long-term nonsense suppression therapy moderates MPS I-H disease progression.

Molecular genetics and metabolism - 1 Mar 2014

Gunn Gwen, Dai Yanying, Du Ming, Belakhov Valery, Kandasamy Jeyakumar, Schoeb Trenton R, Baasov Timor, Bedwell David M, Keeling Kim M

Abstract excerpt

Nonsense suppression therapy is a therapeutic approach aimed at treating genetic diseases caused by in-frame premature termination codons (PTCs; also commonly known as nonsense mutations). This approach utilizes compounds that suppress translation termination at PTCs, which allows translation to continue and partial levels of deficient protein function to be restored. We hypothesize that suppression therapy can...

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Identifiers

PMID: 24411223
DOI: 10.1016/j.ymgme.2013.12.007
PMCID: PMC3943726

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IDUA

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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