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CRISPR Reverses Huntington’s Disease in Mice

12:14 EDT 19 Jun 2017 | Genetic Engineering News

The potential of genome-editing techniques, such as CRISPR/Cas9 , to alleviate disease burden has ignited the imagination for thousands of researchers looking for new therapeutic strategies. Scientists were very quickly able to show that this gene-altering technique could eliminate disease-causing mutations within a variety of tissues in vitro . More recently, CRISPR is being positioned to help treat patients directly, with clinical trials in humans already under way in China and soon to begin in the U.S. Yet, none of the current trials and little research has been done using the technique on neurodegenerative diseases. Now, a group of investigators led by scientists at Emory University is hoping to open up new avenues of neurodegenerative research and rapidly move toward human trials after the release of their new findings. The research team showed that the CRISPR/Cas9 system could snip part of a gene that produces toxic protein aggregates ...

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