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[caliban]

We can't have missed this, but since I did, just to be sure:


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

Paola Scaffidi & Tom Misteli

Here we show that the cellular disease phenotype is reversible in cells from individuals with HGPS. Introduction of wild-type lamin A protein does not rescue the cellular disease symptoms. 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. Upon splicing correction, HGPS fibroblasts assume normal nuclear morphology, the aberrant nuclear distribution and cellular levels of lamina-associated proteins are rescued, defects in heterochromatin-specific histone modifications are corrected and proper expression of several misregulated genes is reestablished. Our results establish proof of principle for the correction of the premature aging phenotype in individuals with HGPS.

--> http://www.nature.co...abs/nm1204.html

[JonesGuy]

Once again, targeted transgenics seems to be an easy answer, eh? More and more we are reinforced with the idea that transgenics contains a possible answer. More and more (and faster, it seems) it's restricted by legislation.

Congrats to that team, however.