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Splice-switching antisense oligonucleotide (ASO) therapies, which can restore functional proteins by targeting splicing of pre-mRNA from defective genes, have had notable clinical successes. However, identifying genetic variants that could be suitable for such an approach is challenging. Reporting in Nature, Timothy Yu and co-workers now present a framework to systematically address this challenge, using ataxia telangiectasia (A-T) as a model. They develop a splice-switching ASO to treat a single patient with an amenable gene variant, showcasing the framework’s potential.