Gene Editing Reduces Alzheimer's Plaque Precursors

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gene editing technology

ScienceDaily

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Gene editing tool reduces Alzheimer's plaque precursor in mice

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A new gene editing tool that helps cellular machinery skip parts of genes responsible for diseases has been applied to reduce the formation of amyloid-beta plaque precursors in a mouse model of Alzheimer's disease.

The application in live mice shows the improved efficiency of the tool, called SPLICER , over the current standard in gene editing technology, as well as the potential for application in other diseases.

"Exon skipping only works if the resulting protein is still functional, so it can't treat every disease with a genetic basis," says Pablo Perez-Pinera .

"The immediate next step is to look at the safety of removing the targeted exons in these diseases, and make sure we aren't creating a new protein that is toxic or missing a key function.".

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