SKIP-AT: a comprehensive, systematic platform approach for targeted exon skipping of ATM

01.02.2024 bis 31.01.2025

We will develop and validate a comprehensive platform approach for targeted exon skipping of constitutive in-frame ATM exons, directly leveraging and extending our established, scalable platform for the development of mutation-specific n-of1/n-of-few ASOs for ATM - with already proven efficacy from preclinical to clinical translation in our hands. Our ATM exon-skipping approach will demonstrate and validate a systematic experimental path combining in silico and in vitro techniques providing a comprehensive assessment to the genomic medicine field on which constitutive exons of the ATM gene can be (ASO-)skipped - and go further by already preparing the most promising skippable exons for clinical ASO development, thus directly paving the way for a novel kind of targeted molecular therapies for A-T.