AgingResearch.NewsJoin free

Modified oligonucleotide targeted to the activated cryptic splice site

Intervention

1 paper4 findings

C, W, M, H: cells, worms or flies, mice, humans. Dark: independent labs found the same change there. Light: one lab or one study. Outline: nothing yet.

Labs are independent when their papers share no last author and no institution.

Against: findings that point the other way from most findings on the same outcome.

On aging: the drug’s trials ARN judged to be about aging, of every trial that tests it for any use.

No results posted: trials that ended more than a year ago with no results on ClinicalTrials.gov and no paper in ARN’s record.

Company ties: findings with an author at a company, or from a paper whose competing-interests statement declares ties to one.

  • 1 lab in 1 country
  • Funder: National Cancer Institute
  • Cells1

Outcomes

4

Heterochromatin-specific histone modifications

upin human cells1

1 study

  1. Modified oligonucleotide targeted to the activated cryptic splice site improves heterochromatin-specific histone modifications in human cells.

    “defects in heterochromatin-specific histone modifications are corrected”

    Cellsin HGPS fibroblasts

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

    Nature medicine6 Mar 2005

More on Heterochromatin-specific histone modifications

Cellular levels of lamina-associated proteins

upin human cells1

1 study

  1. Modified oligonucleotide targeted to the activated cryptic splice site improves cellular levels of lamina-associated proteins in human cells.

    “the aberrant nuclear distribution and cellular levels of lamina-associated proteins are rescued”

    Cellsin HGPS fibroblasts

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

    Nature medicine6 Mar 2005

More on Cellular levels of lamina-associated proteins

Nuclear morphology

upin human cells1

1 study

  1. Modified oligonucleotide targeted to the activated cryptic splice site improves nuclear morphology in human cells.

    “Upon splicing correction, HGPS fibroblasts assume normal nuclear morphology”

    Cellsin HGPS fibroblasts

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

    Nature medicine6 Mar 2005

Mutant LMNA mRNA

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

More on Mutant LMNA mRNA

Latest

Related