Targeted repairs with ‘nicks’ of single DNA strands provide the foundation for novel disease therapies. One of the great challenges of modern medicine is curing debilitating genetic diseases. The development of CRISPR technologies and advancements in genetics research over the past decade has bro
Restorative gene editing using sequences from the counterpart chromosome: The standard CRISPR enzyme Cas9 offers the ability to make repairs but also potentially results in unintended mutations at the targeted site and possibly elsewhere in the genome . In contrast, the nickase enzyme results in more efficient gene correction and no mutagenic events . Credit: Guichard/Bierstrands provide the foundation for novel disease therapies.
Now a team of biologists has developed a new, safer approach that may correct genetic defects in the future. Their strategy makes use of natural DNA repair machinery and provides a foundation for novel gene therapy strategies with the potential to cure a large spectrum of genetic diseases.
“The healthy variant can be used by the cell’s repair machinery to correct the defective mutation after cutting the mutant DNA,” said Guichard, the senior author of the study, “Remarkably, this can be achieved even more efficiently by a simple harmless nick.” Then the researchers tested their new system with Cas9 variants known as “nickases” that targeted just one strand of DNA instead of both. Surprisingly, the authors discovered that such nicks also gave rise to high-level restoration of red eye color nearly on par with normal healthy flies. They found a 50-70% repair success rate with the nickase compared with just 20-30% in dual-strand cutting Cas9, which also generates frequent mutations and targets other sites throughout the genome .
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