Deficiency of U4atac snRNA secondarily results in ciliary defects
Deepak Khatri, Audrey Putoux, Audric Cologne, Sophie Kaltenbach, Alicia Besson, Eloïse Bertiaux, Justine Guguin, Adèle Fendler, Marie Alice Dupont, Clara Benoit‐Pilven, Leila Qebibo, Samira Ahmed-Elie, Séverine Audebert‐Bellanger, Pierre Blanc, Thomas Rambaud, , Marion Delous
The paper identifies a mutation in RNU4ATAC causing ciliary defects in patients with TALS/RFMN/LWS and Joubert syndrome, linking minor splicing defects to ciliary dysfunction.
Proceedings of the National Academy of Sciences · Feb 21, 2023Peer-reviewedGenetic & Genomic Medicine