Efficient genome editing in the mouse brain by local delivery of engineered Cas9 ribonucleoprotein complexes
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Improvements to a gene-editing technology could lead to safe and targeted treatments for neurological diseases.
The gene-editing technology, developed by a team that included researchers from Roche Pharma Research and Early Development, employs Cas9, a DNA-cleaving enzyme. Cas9, which carries an RNA guide that directs it to the specific DNA location in need of editing, was bound on either end by viral tags that allow it to penetrate cell membranes and then enter the nucleus.
The team successfully tested the technology in mice, and found that it could target a variety of specific nerve cells in various regions of the brain without eliciting a significant immune response.
Although it’s not exactly clear how the technology targets specific nerve types, the team believes it shows potential for safely and effectively treating genetic neurological diseases that involve distinct groups of neurons.
- Nature Biotechnology 35, 431–434 (2017). doi: 10.1038/nbt.3806