A Review of CRISPR Tools for Treating Usher Syndrome: Applicability, Safety, Efficiency, and In Vivo Delivery

CRISPR/Cas9 cuts both strands of the DNA, while prime editing cuts only one strand, to repair genetic mutations. They each use different enzymes to make the cuts and the repairs. Base editing converts one letter of the DNA to another letter without cutting the DNA  

The paper compares these three tools and how they can be applied to treat Usher syndrome. The researchers hypothesize that CRISPR/Cas9 can target some types of mutations that cause Usher syndrome but not all of them. Base editing and prime editing can target more types of mutations, but they are newer technologies with unknown safety profiles and delivery challenges, respectively. Further research is suggested to improve the tools and delivery methods to be able to target more mutations, and ultimately, to treat more patients. A key consideration would be ensuring that gene editing tools are safe and do not cause off-target effects or errors in the DNA.

What this means for Usher syndrome: Gene editing tools are promising for treating Usher syndrome, but they require more research and development before they can be used in humans.

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