The role of Musashi-1 in CEP290 c.2991+1655A>G cryptic exon splicing in Leber Congenital Amaurosis
D. Ottaviani, A. Lane, K. Jovanovic, J. C. Gardner, P. E. Sladen, K. L. Hau, A. Brugulat Panes, R. Guarascio, A. J. Hardcastle, M. E. Cheetham
This study investigates the role of Musashi-1 in alternative splicing of CEP290 in human retinal organoids and finds it does not drive the inclusion of the LCA10-associated cryptic exon.
bioRxiv · Aug 4, 2021 · PreprintCell Biology