HIH News

A small molecule has great potential for gene therapy

A small piece of nucleic acid could be the key to success: With its help, a Tübingen research team has succeeded in repairing a gene defect directly in the petri dish. The molecule prevented the nerve cells from producing a defective protein which leads to lethal damages to the cells.

With their proof-of-concept study, the team led by Professor Dr. Ludger Schöls and Dr. Stefan Hauser of the Hertie Institute for Clinical Brain Research, the German Center for Neurodegenerative Diseases and the University of Tübingen has come one step closer to gene therapy for certain forms of ataxia and other dominantly heritable neurodegenerative diseases. The researchers have published their results in the current print issue of the journal "Molecular Therapy - Nucleic Acids". They are offering lab visits to interested media representatives as part of Brain Awareness Week from March 14 - 20, 2022.

Press release (in German only)

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