CRISPR-Cas9 is a prevalent genome-editing technique utilized for investigating specific genes and modifying genes related to diseases. However, it comes with drawbacks such as unintended mutations and toxicity, necessitating the development of a technology that minimizes these side effects to enhanc
in central Japan have now created an enhanced genome-editing approach that significantly reduces mutations, paving the way for more effective treatment of genetic disorders with reduced undesired mutations. Their research has been published inGenome-editing technology centered on CRISPR-Cas9 has revolutionized the food and medicine industries. In the technology, Cas9 nuclease, an enzyme that cuts that guides the enzyme to the required location.
To test this hypothesis, a group consisting of Assistant Professor Masaki Kawamata at Kyushu University and Professor Hiroshi Suzuki at theGraduate School of Medicine constructed a system called “AIMS” in mouse cells, which evaluated the activity of Cas9 separately for each chromosome. Their results showed that the commonly used method was associated with very high editing activity.
To test its effectiveness in a medical setting, they investigated a rare disease called fibrodysplasia ossificans progressiva. Using a mouse model, they were able to create the same genotype as the human version of the disease. Then, using patient-derived iPS cells, they were able to precisely repair damage down to a single nucleotide specifically in the disease-associated allele causing the disease, demonstrating their technique’s usefulness as a safe and efficient gene therapy method.
“We established a new genome editing platform that can maximize the desired editing efficiency by developing activity-regulating [C]gRNAs with appropriate Cas9 activity. Furthermore, we found that ‘safeguard gRNA’ can be applied to various CRISPR tools that require gRNAs by regulating their activities, such as those using Cas12a, which has a different DNA cleavage mechanism,” said Professor Suzuki.
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