A deep intronic mutation causes RAD50 deficiency through an unusual mechanism of distant exon activation.

Bousset, Kristine; Donega, Stefano; Ameziane, Najim; Fleischhammer, Tabea; Ramachandran, Dhanya; Poley-Gil, Miriam; Schindler, Detlev; van de Laar, Ingrid M et al. · J Clin Invest · 2024

basic_science · Level V

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Abstract

This study identifies and characterizes a novel type of splicing mutation in RAD50 deficiency, a rare genetic disorder.

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