New Gene Editing Tool for Simultaneous Multiple Edits Developed
Scientists develop a new biotechnology tool to edit DNA at multiple sites simultaneously and error-free.
A crafty representation of gene editing. Researchers developed a new gene editing tool for multiple simultaneous edits.
- The human genome consists of around 3 billion base pairs and humans are all 99.6% identical in their genetic makeup, with the small 0.4% accounting for any difference between one person and another.
- Specific combinations of mutations in those base pairs hold important clues about the causes of complex health issues, including heart disease and neurodegenerative diseases like schizophrenia.
- Researchers from the University of California San Diego have developed new genome editing tools called multiplexed orthogonal base editors (MOBEs) to install multiple point mutations at once.
- MOBEs use RNA structures called aptamers to recruit base-modifying enzymes to specific genomic locations, enabling simultaneous editing of multiple sites with high efficiency and a lower incidence of crosstalk.
- The study was a proof of principle to test the feasibility of the MOBE system, which has been granted a provisional patent.
- To test them even further, the team conducted several case studies with real diseases, including Kallmann syndrome, a rare hormonal disorder.
- The researchers hope that other scientists will use the MOBEs to model genetic diseases, learn how they manifest and then hopefully create effective therapies.