The scientist rewriting DNA, and the future of medicine
- David Liu, an American molecular biologist, was awarded a $3 million Breakthrough Prize for developing two gene editing technologies, base editing and prime editing.
- Base editing allows correcting around 30% of mutations causing genetic diseases, and is already in 14 clinical trials, including treating patients with a lung and liver disorder.
- Prime editing, introduced in 2019, can replace entire faulty DNA sequences, making it 'the most versatile way we know of to edit the human genome' that could treat genetic diseases.
83 Articles
83 Articles
Increasing Cas9-mediated homology-directed repair efficiency through covalent tethering of DNA repair template
The CRISPR-Cas9 system is a powerful genome-editing tool in which a guide RNA targets Cas9 to a site in the genome, where the Cas9 nuclease then induces a double-stranded break (DSB). The potential of CRISPR-Cas9 to deliver precise genome editing is hindered by the low efficiency of homology-directed repair (HDR), which is required to incorporate a donor DNA template encoding desired genome edits near the DSB. We present a strategy to enhance HD…
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