Head
|
ASO4Kids: Establishing a pipeline for the development of allele-selective antisense oligonucleotide therapies for rare neurological disorders
Funding line:
Else Kröner Excellence Fellowships
Dr. Matias Wagner und team analysing data.
(© Kathrin Czoppelt, TUM university hospital)
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.
Further information: https://humangenetik.mri.tum.de/en/ag-nucleic-acid-therapies-dr-matias-wagner