Ratti, A. & Buratti, E. Physiological functions and pathobiology of TDP-43 and FUS/TLS proteins. J. Neurochem. 138, 95–111 (2016).
Guo, L. & Shorter, J. Biology and pathobiology of TDP-43 and emergent therapeutic strategies. Cold Spring Harb. Perspect. Med. 7, 024554 (2017).
Niksic, M., Romano, M., Buratti, E., Pagani, F. & Baralle, F. E. Functional analysis of cis-acting elements regulating the alternative splicing of human CFTR exon 9. Hum. Mol. Genet. 8, 2339–2349 (1999).
Pagani, F. et al. Splicing factors induce cystic fibrosis transmembrane regulator exon 9 skipping through a nonevolutionary conserved intronic…
