HIH News

Important step towards personalized treatment for Parkinson's disease

In a recent publication, an international consortium led by the „Luxembourg Centre for Systems Biomedicine“ shows how the serious consequences of a rare mutation in Parkinson's disease can be precisely counterbalanced – although so far only in the laboratory. Professor Thomas Gasser from the Hertie Institute for Clinical Brain Research contributed to the research.

In a recent publication, an international consortium led by the „Luxembourg Centre for Systems Biomedicine“ shows how the serious consequences of a rare mutation in Parkinson's disease can be precisely counterbalanced – although so far only in the laboratory. Professor Thomas Gasser from the Hertie Institute for Clinical Brain Research contributed to the research.

The rare mutation causes early-onset Parkinson’s. The researchers were able to show that the mutation leads to a truncated version of an important RNA. Patients carrying this mutation do not produce a smaller version of the corresponding protein which might still be functional, but completely fail to synthesize the protein, which is critically involved in energy metabolism. As a result, the nerve cells die. The researches detected substances which were able to correct the RNA-defect and thus compensate for the protein loss.  The results are an important step towards a personalized therapy for patients with Parkinson’s disease carrying this particular mutation.

Link to the Press Release of the Luxembourg Centre for Systems Biomedicine of the University of Luxembourg: https://wwwen.uni.lu/university/news/slideshow/new_approach_against_parkinson_s_disease_through_stem_cell_research

 

Original Publication:

Boussaad I; Obermaier CD et al. A patient-based model of RNA mis-splicing uncovers treatment targets in Parkinson’s disease. Science Translational Medicine, 9. September 2020.

DOI: 10.1126/scitranslmed.aau3960

 

Contact:

Professor Thomas Gasser

Center of Neurology
Hertie Institute for Clinical Brain Research (HIH)
Department Neurodegenerative Diseases

Hoppe-Seyler-Straße 3
72076 Tübingen

Tel.: +49 (0)7071 29-86529

thomas.gasseruni-tuebingen.de

 

Communications

Dr. Mareike Kardinal (on parental leave)
Director of Communications
Hertie Institute for Clinical Brain Research
Otfried-Müller-Str. 27
72076 Tübingen

 

Stand-in:

Dr. Hildegard Kaulen
Karlsruher Str. 8
65205 Wiesbaden

Tel: 06122 52718
h.k[at]kaulen-wissenschaft.de