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ASO4Kids: Establishing a pipeline for the development of allele-selective antisense oligonucleotide therapies for rare neurological disorders

Institution: Institute of Human Genetics, TUM School of Medicine & Health, Technical University of Munich (TUM).
Applicant: Dr. Matias Wagner
Funding line:
Else Kröner Excellence Fellowships
ASO4Kids: Establishing a pipeline for the development of allele-selective antisense oligonucleotide therapies for rare neurological disorders

The ASO4Kids project aims to develop new treatments for children with rare genetic brain disorders that lead to severe epilepsy and developmental delays. Dr. Matias Wagner is building an efficient development platform for antisense oligonucleotides (ASOs) – short strands of synthetic DNA or RNA to switch off specific disease-relevant genetic signals. By targeting common genetic markers, a small number of customized treatments are to be designed that can benefit as many affected patients as possible. In the long term, this platform seeks to accelerate the development of personalized treatments and significantly improve their availability.