"Molecular bullets" against severe, rare, genetic diseases are developed at ICGEB

Researchers at ICGEB Trieste have their finger on the pulse of therapeutic strategies for Cystic Fibrosis, Hemophilia and Spinal Muscular Atrophy

The molecular bullets, which are used to remedy "oversights" in cellular mechanisms responsible for synthesizing proteins, are the subject of the recently published scientific article in Human Molecular Genetics (Oxford Journals), available in Open Source.

Franco Pagani, Group Leader of the Human Molecular Genetics lab at ICGEB Trieste, together with his team, has proven the potential of a therapeutic strategy based on small RNAs in treating three serious genetic diseases: cystic fibrosis, spinal muscular atrophy (SMA) and hemophilia; an approach that might well extend to other diseases in the near future.

The study, which demonstrates the potential of a novel therapeutic strategy using small RNAs (EXSpeU1s) active in the correction of defects found in splicing, has been financed by Telethon and the Italian Cystic Fibrosis Research Foundation. The study, undertaken in collaboration with the University of Ferrara, Italy, has led to the development of a new patent, the latest in a string of 58 currently active ICGEB patents.

ICGEB is also funded by the Italian Ministry of Foreign Affairs.


Further reading:

Press Release (in Italian): Rare and infectious genetic diseases: ICGEB, excellence in basic research in Italy. 

Telethon.it : Molecular Bullets on the way for Cystic Fibrosis, SMA and Haemophilia

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