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Ray of hope for human Usher syndrome patients
Date:12/4/2012

that the active gene product was synthesized in the cell and organ cultures. Both active substances, PTC124 and NB54, generally enhanced read-through efficacy and exhibited improved tolerability in mouse and human retinal cultures in comparison with clinically employed antibiotics. The team also successfully documented read-through of the mutation in vivo a mouse model.

"Our gene-based treatment strategies, involving gene repair as well as read-through therapy, represent valuable and promising alternatives to viral gene addition and may actually be the only treatment option for the large and isoform-rich USH genes. We hope that these alternatives will make a significant contribution to the therapy of both Usher syndrome patients as well as others with severe genetic retinal pathologies and other genetic disorders," explains Dr. Kerstin Nagel-Wolfrum.

In addition to continuing its preclinical studies into the use of the active substances, the Mainz Usher research team plans to make its new Usher syndrome therapy available to patients as soon as possible.


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Contact: Dr. Kerstin Nagel-Wolfrum
nagelwol@uni-mainz.de
49-613-139-20131
Johannes Gutenberg Universitaet Mainz
Source:Eurekalert

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