The project by Gambari et al. proposes to identify miRNAs that regulate (i) CFTR, (ii) transcription factors that regulate CFTR, and (iii) factors that enhance CFTR cell surface expression. Next, they will design and synthesize PNAs and PNA masks to inhibit these miRNAs and their interactions with specific mRNAs with important roles in CF, respectively; the effect of these PNA molecules and combinations thereof on gene expression and the secretome will be assessed. MiRNAs in various CF body fluids will be quantified. Overall the project aims to develop new therapeutics to enhance CFTR expression/function that may be used as adjunct therapies for people with CF.
Revealing the microRNA-transcription factors network in cystic fibrosis:from microRNA therapeutics to precision medicine (CF-miRNA-THER) FFC 7/2018
Gambari R;Cabrini G;
2018
Abstract
The project by Gambari et al. proposes to identify miRNAs that regulate (i) CFTR, (ii) transcription factors that regulate CFTR, and (iii) factors that enhance CFTR cell surface expression. Next, they will design and synthesize PNAs and PNA masks to inhibit these miRNAs and their interactions with specific mRNAs with important roles in CF, respectively; the effect of these PNA molecules and combinations thereof on gene expression and the secretome will be assessed. MiRNAs in various CF body fluids will be quantified. Overall the project aims to develop new therapeutics to enhance CFTR expression/function that may be used as adjunct therapies for people with CF.I documenti in SFERA sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.