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Research Article – Effective gene therapy in an authentic model of Tay-Sachs-related diseases

Although this research article was published in 2006 we thought it was important that the findings in the study titled “Effective gene therapy in an authentic model of Tay-Sachs related diseases” were highlighted once again. The article was written by M. Begoña Cachón-González, Susan Z. Wang, Andrew Lynch, Robin Ziegler, Seng H. Cheng and Timothy M. Cox and was published in PNAS July 5, 2006 vol. 103 no. 27.

As THE CATS FOUNDATION readership increases it is important that people are able to read about the research which has been completed both in the past and more recently.

The research article

To read the article you can either click on the image below or access it online here.

Effective gene therapy in an authentic model of Tay-Sachs-related diseases

During the study, Cachón-González et al (2006) found that when mice with authentically modelled Tay-Sachs and the related Sandhoff disease (both are part of the GM2 gangliosidosis family) were administered a single treatment in early adult life there was an increased survival rate. In addition, these amazing results showed that the treatment prevented the onset of the devastating neurological disease that mimics the acute illness occurring in babies and young children.

Source: M. Begoña Cachón-González, Susan Z. Wang, Andrew Lynch, Robin Ziegler, Seng H. Cheng and Timothy M. Cox (2006), Effective gene therapy in an authentic model of Tay-Sachs-related diseases. PNAS July 5, 2006 vol. 103 no. 27.

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