Tuesday, March 8, 2005Genetic abnormalities behind the premature aging disease Progeria have been corrected in cells in a step towards gene therapy for the condition.
Affecting about one in eight million children, Hutchinson-Gilford Progeria (HGP) causes sufferers to rapidly experience symptoms of old age.
The average life expectancy of those afflicted is just around 14 years, with many dying from such age-related conditions as heart disease.
The disease is known to be caused by a mutation in the gene lamin A.
The mutation is thought to adversely affect the expression of genes by preventing appropriate genetic “editing.”
The inappropriate editing cuts exons—”sentences” of genetic information—too short.
Tom Misteli and colleagues of the US National Cancer Institute have now found a way to correct the editing error.
They did this using synthetic molecules called morpholino oligonucleotides that Misteli likens to a “molecular Band-Aid.”
The researchers say the discovery could aid the development of gene therapy for Progeria.
The research is reported in the journal Nature Medicine.
- Paola Scaffidi & Tom Misteli. “Reversal of the cellular phenotype in the premature aging disease Hutchinson-Gilford progeria syndrome” — Nature Medicine, March 6, 2005
- “Ageing disease ‘gene clue’ found” — BBC News, March 7, 2004
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