Acknowledgements 
F.P. is listed as inventor of the US patent n. 9 669 109 ‘A modified human U1snRNA molecule, a gene encoding for the modified human U1snRNA molecule, an expression vector including the gene, and the use thereof in gene therapy of familial dysautonomia and spinal muscular atrophy’. As such the inventors could potentially benefit from any future commercial exploitation of patent rights, including the use of ExspeU1s in CF.