Scientists Target Incurable Mitochondrial Diseases With New Gene Editing Tools
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Summary
A team from the University of Washington in Seattle has used a gene editing tool called base editing to successfully target and rectify mitochondrial DNA mutations in mice, thereby overcoming the organelles’ deadly mutations.
While the team admits that there is “more work to do”, the early results suggest that DNA mutations in mitochondria damage cells’ ability to produce energy and that correcting such mutations could prove beneficial.
Furthermore, the team’s new mitochondrial base editor could improve the accuracy of mitochondrial gene therapy.
The base editor has already been used to successfully reprogram mitochondria in cultured human cells.
After being injected into the bloodstreams of young adult mice, it reached the cells of their hearts and muscles, more than doubling normal transporter molecule levels in cardiac cells.
However, the brains and kidneys were not reached, and very few liver cells were affected.