Luís Pereira

Sandro Alves

Portuguese, Swiss and French researchers show, for the first time, that is possible to inhibit, in a living organism, the mutated copies of a gene without affecting any existing normal copies of the same gene. The research, to appear in the 8th of October edition of the journal PLoS One, describes how scientists successfully used the approach in rats to reverse the symptoms of Machado Joseph Disease (MJD), an untreatable and potentially fatal neurodegenerative disease. If these results can be transferred to humans – and the method is shown to work on isolated human cells - it can, not only become the first available treatment for MJD, but also open the door to promising new safer and more efficient gene therapy for other neurodegenerative disorders, such as Alzheimer’s or Parkinson’s disease.

* This article is the result of a project for the divulgation of portuguese science" http://www.cienciahoje.pt/1455"