SKIP-AT: a comprehensive, systematic platform approach for targeted exon skipping of ATM
Awardee: Matthis Synofzik
Institution: Hertie-Institute for Clinical Brain Research, University of Tübingen, Germany
Grant Amount: $53,240.00
Funding Period: February 1, 2024 - January 31, 2025
Summary:
We will develop and validate a systematic, comprehensive platform approach for targeted exon skipping of constitutive ATM exons. For this, we can directly leverage and extend our established, scalable platform for the development of patient-specific antisense oligonucleotides (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 that will allow to provide the genomic medicine field with the urgently warranted comprehensive knowledge on which exons of the ATM gene can be (ASO-)skipped - and in fact even allows to already prepare the most promising skippable exons for clinical ASO treatment development.