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PTBP1 acts as a dominant repressor of the aberrant tissue‐specific splicing of ISCU in hereditary myopathy with lactic acidosis

BACKGROUND: Hereditary myopathy with lactic acidosis (HML) is an autosomal recessive disease caused by an intron mutation in the iron‐sulfur cluster assembly (ISCU) gene. The mutation results in aberrant splicing, where part of the intron is retained in the final mRNA transcript, giving rise to a tr...

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Detalhes bibliográficos
Publicado no:Mol Genet Genomic Med
Main Authors: Rawcliffe, Denise F. R., Österman, Lennart, Nordin, Angelica, Holmberg, Monica
Formato: Artigo
Idioma:Inglês
Publicado em: John Wiley and Sons Inc. 2018
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6305642/
https://ncbi.nlm.nih.gov/pubmed/30209894
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/mgg3.413
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