Saturation genome editing ofRNU4-2reveals distinct dominant and recessive neurodevelopmental disorders

De Jonghe J., Kim HC., Adedeji A., Leitão E., Dawes R., Chen Y., Blakes AJM., Simons C., Rius R., Alvi JR., Amblard F., Austin-Tse C., Baer S., Balton EV., Blanc P., Calame DG., Coutton C., Cunningham CA., Dargie N., Dipple KM., Du H., El Chehadeh S., Glass I., Gleeson JG., Grunewald O., Gueguen P., Harbuz R., Jacquemont M-L., Leventer RJ., Marijon P., Messaoud O., Sultan T., Thauvin C., Vincent-Delorme C., Yilmaz Gulec E., Thevenon J., Mendez R., MacArthur DG., Depienne C., Nava C., Whiffin N., Findlay GM.

DOI

10.1101/2025.04.08.25325442

Type

Preprint

Publication Date

11/04/2025

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