A study published in the scientific journal Science Translational Medicine (here) reports further progress in the development of gene therapy strategies for cystic fibrosis, particularly for forms that currently have no effective treatments. Among the authors is Anna Cereseto, a researcher supported by our Foundation.

The findings show that RNA-mediated gene therapy approaches may overcome specific CFTR gene mutations, restoring protein function in experimental systems relevant to the disease. This work fits within a key strategic area in which the Foundation is strongly engaged: the development of effective delivery systems for gene therapy technologies targeting lung cells. Our strategic project GenDel-CF is part of this effort.

Another important aspect of this research is the use of respiratory cells derived from people with cystic fibrosis, which allow the effectiveness of new strategies to be tested in highly representative disease models. The FFC Ricerca Primary Cell Culture Service actively contributes to these studies, supporting the development of increasingly targeted approaches.

Overall, these results reinforce the value of the Foundation’s investment in innovative projects aimed at developing therapeutic solutions for all people with cystic fibrosis, especially those who still lack effective options.

This topic will also be discussed during our Spring Seminar on May 16: check the program and join us online (event in Italian).