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Hope for children with rare genetic defect
Date:4/19/2011

lization of the protein networks is possible with the help of the Multi-Epitope-Ligand Cartography (MELC), an innovative microscopy technology that produces three-dimensional "snapshots" of the arrangement of dozens of proteins in the cell. "We now know, for example, that proteins that would never meet in a healthy nervous cell have contact in JNCL, but we also know that contacts get lost due to the disease," states Anton Petcherski. During his master's thesis in Dr. Ruonala's working group, he used MELC and found several peculiarities in the protein constellation of cells from brains of transgenic mice that mimic the human JNCL. He then defined the particularly significant "biomarkers" that now serve as the basis of this screening.

At Harvard Medical School in Boston, the group of Dr. Susan Cotman has been occupied with JNCL for several years. Dr. Cotman is the "mother" of the genetically precise JNCL mouse model that the Frankfurt group uses. Dr. Cotman's working group had already carried out a high throughput screening for the active ingredients for the treatment of the disease back then, however, only a few biomarkers were known. "Since there are many anomalies in the cooperation of the proteins in the case of JNCL, the disease pattern does not necessarily improve when we are able to influence one area in a correcting manner," says Ruonala. "It is also a question of hierarchy in the protein network. Some proteins are more important for the cohesion of the network than others."

Now the two working groups are going to join forces: As part of his doctoral thesis, Anton Petcherski will be going to Boston for one year in order to search for a suitable medication for JNCL in the local drug collections. The researchers limit their search to already approved drugs, since this considerably shortens the transfer of potential medication to clinical trial. In their own laboratory, the Frankfurt group already found one active ingredient that inhibits the
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Contact: Dr. Mika Ruonala
Ruonala@em.uni-frankfurt.de
49-697-982-9463
Goethe University Frankfurt
Source:Eurekalert  

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