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

Basic data

Title:
SKIP-AT: a comprehensive, systematic platform approach for targeted exon skipping of ATM
Duration:
01/02/2024 to 31/01/2025
Abstract / short description:
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.
Keywords:
ataxia
Ataxie
gene therapy
Gentherapie
RNA-Therapie
Antisense Oligonukleotide
Ataxia teleangiectasia

Involved staff

Managers

Faculty of Medicine
University of Tübingen
University Department of Neurology
Hospitals and clinical institutes, Faculty of Medicine

Local organizational units

Department of Neurology with Focus on Neurodegenerative Disorders
University Department of Neurology
Hospitals and clinical institutes, Faculty of Medicine
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