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An Exon-Specific Small Nuclear U1 RNA (ExSpeU1) Improves Hepatic OTC Expression in a Splicing-Defective spf/ash Mouse Model of Ornithine Transcarbamylase Deficiency

OTC splicing mutations are generally associated with the severest and early disease onset of ornithine transcarbamylase deficiency (OTCD), the most common urea cycle disorder. Noticeably, splicing defects can be rescued by spliceosomal U1snRNA variants, which showed their efficacy in cellular and an...

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Detalhes bibliográficos
Publicado no:Int J Mol Sci
Main Authors: Balestra, Dario, Ferrarese, Mattia, Lombardi, Silvia, Ziliotto, Nicole, Branchini, Alessio, Petersen, Naomi, Bosma, Piter, Pinotti, Mirko, van de Graaf, Stan F. J.
Formato: Artigo
Idioma:Inglês
Publicado em: MDPI 2020
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Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7699343/
https://ncbi.nlm.nih.gov/pubmed/33228018
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms21228735
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