Long-Term Nonsense Suppression Therapy Moderates MPS I-H Disease Progression

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 con...

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Autors principals: Gunn, Gwen, Dai, Yanying, Du, Ming, Belakhov, Valery, Kandasamy, Jeyakumar, Schoeb, Trenton R., Baasov, Timor, Bedwell, David M., Keeling, Kim M.
Format: Artigo
Idioma:Inglês
Publicat: 2013
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Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3943726/
https://ncbi.nlm.nih.gov/pubmed/24411223
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2013.12.007
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