The project is part of the research field aimed at assisting and enhancing the effectiveness of CFTR modulation treatments, which still have action limits. Here, microRNAs (miRNAs), which are small molecules of RNA (ribonucleic acid) active in the cell cytoplasm and capable of influencing, as “transcription factors”, the expression and the membrane stability of the CFTR protein, are involved, interfering in the so-called “transcription”. This characterizes the transfer of the genetic code from the DNA to the messenger RNA (mRNA) for the synthesis of the CFTR and other proteins that collaborate in its maturation and stability. This influence is generally exercised by limiting the transcription and therefore the final expression of the protein. The study is aimed at the identification of particular miRNAs (and related target mRNAs) with the aim of modifying the gene expression of cells from cystic fibrosis patients and, in particular, increase the stability/expression of the CFTR protein. The contribution of therapeutic effect that the modulation of critical miRNAs (by some peptidonucleic acids) may have in combination with the currently used CFTR modulators will also be tested.
WHO ADOPTED THE PROJECT
€ 39.000
€ 20.000
Delegazione FFC di Como Dongo
€ 17.000