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Mutant LMNA mRNA

Biomarker

1 paper1 finding

  • Cells1

Interventions

1

Modified oligonucleotide targeted to the activated cryptic splice site

downin human cells1

1 study

  1. Modified oligonucleotide targeted to the activated cryptic splice site decreases mutant LMNA mRNA in human cells.

    “The mutant LMNA mRNA and lamin A protein can be efficiently eliminated by correction of the aberrant splicing event using a modified oligonucleotide targeted to the activated cryptic splice site.”

    Cellsin HGPS fibroblasts

    Reversal of the cellular phenotype in the premature aging disease Hutchinson-Gilford progeria syndrome

    Nature medicine6 Mar 2005

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