Premature aging disease reversed in cells

March 8, 2005 Genetic 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&mdash;"sentences" of genetic information&mdash;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.