An antisense oligonucleotide-based strategy to ameliorate cognitive dysfunction in the 22q11.2 Deletion Syndrome
Pratibha Thakur, Martin Lackinger, Anastasia Diamantopoulou, Sneha B. Rao, Yijing Chen, Khakima Khalizova, Annie Ferng, Curt Mazur, Holly B. Kordasiewicz, Robert J. Shprintzen, Sander Markx, Bin Xu, Joseph A. Gogos
This study shows that reducing EMC10 levels in 22q11.2 deletion syndrome models improves cognitive function and sustains benefits for over two months.
eLife · Apr 17, 2025Peer-reviewedNeuroscience