From cancer to Alzheimer’s: here are the prospects for gene silencing.

A discovery that has been compared to that of penicillin: Rnai (RNA interference) – awarded the Nobel Prize in 2006 – has become a new class of innovative medications, today for rare genetic diseases, in the future for common diseases such as cancer and Alzheimer.

Rnai is a natural mechanism which allows, if used as a therapy, to silence messenger RNAs (mRNAs), that is the genetic precursors that code for the proteins that cause disease, and to prevent, or significantly limit their translation into proteins. An innovative approach, which aims therefore at the “root” of the disease rather than its symptoms.

«Thanks to the siRna (small interfering Rna) and to the antisense Rna many pathologies are being fought rare, but also diseases with wider diffusion, and one day also in the treatment of Alzheimer’s and Huntington’s diseases – explains Stefano Vella, Adjunct Professor of Global Health, Catholic University of the Sacred Heart – The hope is that yes may in the future use RNA for treat cancer, which is now being treated with drugs that block “bad” molecules, but are unable to penetrate anywhere in the cells, as RNA can, blocking the production of these molecules to the root. The turning point would be epochal. We know that there are many common diseases that are caused, at least in part, by genetic abnormalities. This is true for diabetes, arterial hypertension and even Alzheimer’s».

«One of the characteristics that explains why RNA is such a promising and interesting approach is the speed with which we can create drugs. Self in fact we know the gene we want to silence, we can look up a computer the sequence of that gene and create the small Rna molecule specific interferent (siRna). We can do it in a few weeks, here in the lab. And in a few months we can test the drug on animals. In a couple of years, then, we usually manage to pass the phase conceptual to clinical phase 1. It is quite rare to be able to generate drugs this way, so quickly and so reliably. But we and others have proven that with Rnai this is possible» – said Akshay Vaishnaw, president of Alnylam Pharmaceuticals, the US biotech that in just two years launched on the Italian market three innovative RNAi drugs approved by AIFA»

Lavoro realizzato da Maria Catena e Mattia Ceraso

IV B Classico I.I.S. Sant’Agata M.llo “L.Sciascia”.