Back

Ciz1-Loss Causes Female-Specific Autosomal Neurodevelopmental Disorder Through Defective X-Inactivation Maintenance

Besnard, T.; Loda, A.; Kneuss, E.; Do Souto Ferreira, L.; Ebstein, F.; Vignard, V.; Deb, W.; Küry, S.; Derradji-Costea, M. M.; Landeau-Trottier, G.; Talarmain, P.; Trochu, E.; Dutan Patino, V.; Piton, A.; Lamouche, J.-B.; Gibaud, M.; Behunova, J.; Laccone, F.; Steinkellner, H.; Blanc, X.; Broly, M.; Ranza, E.; Abumansour, I.; Hashem, M. O.; Shamseddin, H. E.; Albarakati, R.; Alfadhel, M.; Oprea, G.; Rad, A.; Abdullah Alabdi, L.; Alhaddad, B.; Bakur, K.; Alanazi, T.; Madani, J.; Ghayoor Karimiani, E.; Houlden, H.; Maroofian, R.; McNee, G. N.; Stewart, G. S.; Antonarakis, S.; Alkuraya, F.; Bezi

2025-12-20 genetic and genomic medicine
10.64898/2025.12.19.25342284 medRxiv
Show abstract

We report an autosomal recessive neurodevelopmental disorder exclusively affecting females carrying biallelic loss-of-function variants in CIZ1, a gene encoding a nuclear matrix protein essential for the maintenance of X-chromosome inactivation. Eight unrelated affected females were identified, whereas male siblings with biallelic variants were asymptomatic. We showed that loss of CIZ1 compromises the maintenance of X-chromosome inactivation, leading to an abnormal overexpression of a subset of X-linked genes.

Matching journals

The top 11 journals account for 50% of the predicted probability mass.

50% of probability mass above

"Similar papers" are the closest papers from that journal in the model's embedding space. They show what the match is built on, but the ranking comes mostly from a classifier over the whole training set, not from these examples alone.