- Publication 1 - Restoration of the cystic fibrosis transmembrane conductance regulator function by splicing modulation
- Publication 2 - Introducing sense into nonsense in treatments of human genetic diseases
- Publication 3 - The unfolded protein response affects readthrough of premature termination codons
- Publication 4 - Take it personally: how personal we reach when we are so different from each other?
- Publication 5 - The suppression of premature termination codons and the repair of splicing mutations in CFTR
- Publication 6 - Antisense oligonucleotide splicing modulation as a novel Cystic Fibrosis therapeutic approach for the W1282X nonsense mutation
- Publication 7 - Delivery Characterization of SPL84 Inhaled Antisense Oligonucleotide Drug for 3849 + 10 kb C- > T Cystic Fibrosis Patients
- Publication 8 - The safety and toxicity profile of SPL84 an inhaled antisense
- Publication 9 - A phase I study assessing the safety and tolerability of SPL84, an inhaled antisense oligonucleotide for treatment of cystic fibrosis patients with the 3849 +10kb C->T
