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Hebrew University develops novel approach for treating mitochondrial disorders
Date:6/30/2008

activity of the LAD enzyme within the mitochondria.

The clinical course of LAD deficiency is variable, presenting in infancy as a neurological disease of varying severity, or later in life with life-threatening recurrent episodes of liver failure. Most LAD deficiency patients suffer from neurological symptoms.

In developing DERT, Rapoport proposed a novel but simple approach for the delivery of an artificial mitochondrial enzyme directly to its sub-cellular location. The approach is to fuse the manufactured LAD enzyme with a specific delivery component, which will then lead the LAD into the cells and their mitochondria. There, it will substitute for the mutated endogenous enzyme.

For the past four years, Rapoport, under the guidance of Hebrew University Prof. Haya Loberboum-Galski, has been experimenting in vitro on cells taken from patients suffering from LAD deficiency. The results have been highly positive. Experiments are also now being conducted on the in-vivo model of this disorder in LAD-deficient mice. Very promising preliminary results show the ability of this therapy to improve LAD enzymatic activity even in the mouse brain.

Rapoport's invention of enzyme replacement therapy for mitochondrial disorders is patented by Yissum, the Technology Transfer Company of the Hebrew University of Jerusalem. This invention possesses great potential to becoming the treatment of choice for various types of mitochondrial disorders and consequently encompasses a significant commercial potential, as today there is no known medical cure.


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Contact: Rebecca Zeffert
rebeccaz@savion.huji.ac.il
972-025-881-641
The Hebrew University of Jerusalem
Source:Eurekalert

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