ProjectTARGET A-T canonical splice: a novel ASO strategy to substantially enlarge the number of A-T patients amenable to…

Basic data

Title:
TARGET A-T canonical splice: a novel ASO strategy to substantially enlarge the number of A-T patients amenable to individualized ASO therapies
Duration:
01/04/2024 to 31/03/2025
Abstract / short description:
Leveraging our established ATM splice modulation antisense oligonucleotide (ASO) platform - already proven successful for targeting cryptic ATM splice variants from preclinical design to clinical translation -, we will here extend this comprehensive, scalable ATM ASO platform by demonstrating and validating a systematic experimental path also for canonical splice-targeting ASO strategies for ATM.
Keywords:
ataxia
Ataxie
rare diseases
seltene Erkrankungen
RNA-Therapie
Neurogenetik
Präzisionsmedizin
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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