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Sleeping Beauty hooks up with herpes to fight brain disease
Date:1/8/2010

for GFP from the herpes package to the genome of the human cells, where the gene was stably expressed.

The team has previously shown that the Sleeping Beauty/herpes combination works efficiently in brain cells known as neural progenitor cells, which go on to form brain cells known as neurons. Modifying these cells perhaps by adding a gene that creates a protein crucial for health is one technique scientists are experimenting with to try to treat several brain diseases that are currently untreatable.

The gene segment used in the experiment described in Gene Therapy was about 12 kilobases long, which is larger than the limit of either AAV (4.5 kb) or lentiviruses (9 kb). Those few kilobases matter, a lot. The ability to transfer bigger genes gives scientists room to try to address more diseases with a gene therapy approach. The added space also makes it possible to include more regulatory elements instructions that help determine how and when genes are turned on or off. This allows scientists to package additional safety directives, in the form of more DNA, along with the gene designed to treat the disease.


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Contact: Tom Rickey
tom_rickey@urmc.rochester.edu
585-275-7954
University of Rochester Medical Center
Source:Eurekalert

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