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Therapeutic challenge in Leukodystrophies: Translational and ethical research towards clinical trials

Therapeutic challenges in leukodystrophies: Translational and ethical research towards clinical trials.

Leukodystrophies (LDs) are inherited rare neurodegenerative diseases of the white matter and its main component, the myelin. LD are affecting predominantly children. The severity of the disease is related to axonal dysfunction due to myelin deficiency or destruction.

Despite the achievement of remarkable advances made in the past decade, there is no current curative therapy. The development of therapeutic approaches for myelin repair and neuroprotection constitutes the main objective of the LeukoTreat project. Indeed, LDs constitute prototypic pathologies to tackle myelin formation/destruction issues as well as glial cell dysfunction in neurodegeneration. The global aim is to promote the development of therapeutic strategies for the largest possible number of LD-affected patients and further applications to more common white matter disorders and finally neurodegenerative diseases.

Researchers of the Medical University

The Medical University of Vienna is project partner under the leadership of Univ.-Prof. Johannes Berger, Center for Brain Research, Department of Pathobiology of the Nervous System.

Univ.-Prof. Johannes Berger

Head of project

Center for Brain Research
Department of Pathobiology of the Nervous System


Project coordinator: Prof. Odile Boespflug-Tanguy, University Paris Diderot, France


Project duration1. March 2010 until 30. September 2013
Funding volume, total€ 5.978.126,00
Funding volume, Medical University of Vienna€ 337.888,00


In the LEUKOTREAT project under the leadership of Univ.-Prof. Dr. Johannes Berger, 24 leading scientists from different countries have united themselves, to find new therapeutic approaches for leukodystrophy treatement. Find out more at the official website!