Abstract Neurodevelopmental disorders (NDDs) affect 2–4% of the population, are predominantly genetic and remain unsolved in ~50% of individuals. We show that rare biallelic variants in RNU2-2 are enriched and over-transmitted in individuals with unresolved NDDs. We define a recessive RNU2-2 syndrome, delineate its unique genetic architecture and show that it manifests clinically as a severe developmental and epileptic encephalopathy. We find that candidate biallelic variants are significantly c
Biallelic variants in RNU2-2 cause a remarkably frequent developmental and epileptic encephalopathy
A. H. JACKSON·Siddharth Banka·Bader Alhaddad·Olivia J. Henry·A M Delgado-Vega·Elizabeth Wall·Ola Abdelhadi·Shakti Agrawal·Khadijah Bakur·Edward Blair·A. F. Brady·Helen Brittain·Kate Chandler·Natasha Clarke·Miriana Danelli·Nicholas Drinkall·Irene Duba·Frances Elmslie·Jamie M. Ellingford·Lisa J. Ewans·Gabriella Gazdagh·Simon P. Heller·Anna Hammarsjö·Kristina Karrman·Usha Kini·Nicole Lesko·Anna Lindstrand·Rebecca Macintosh·Sahar Mansour·Lara Menzies·Kay Metcalfe·Alison Milhench·L. Nashef·Raymond T. O’Keefe·Nadja Pekkola Pacheco·Elizabeth E. Palmer·Amitav Parida·Katrina Prescott·Melody Redman·Alessandra Renieri·Chiara Fallerini·Caterina Lo Rizzo·Rani Sachdev·Cas Simons·Sanjay M. Sisodiya·Helen Stewart·Tommy Stödberg·Benito Banos-Pinero·Fulya Taylan·Helen Thomas·Flavia Tinella·Samuel Agyei Wiafe·Anna Wedell·Andrew Fennell·Susan Walker·Rocío Rius·Jong Hee Chae·Ann Nordgren·Fowzan Alkuraya·Jenny Lord·Nicola Whiffin·Alexander J. M. Blakes
