The CRISPR Toolbox
Can we move beyond Cas9 to reduce unintended off-target effects of CRISPR technology?
Garrett Rettig | | Opinion
Since CRISPR (clustered regularly interspaced short palindromic repeats) gene editing was discovered in 2012, its ability to make precise and permanent changes in the DNA of both animals and plants has been generating excitement. The focus of medical science research to-date has been on diseases caused by a single gene mutation, such as sickle cell anemia (SCA) and beta thalassemia, and the improvement of anti-tumor immunotherapy. The first use of an investigational ex vivo CRISPR-based therapy to treat SCA and beta thalassemia is already underway in at least two patients in clinical trials.
CRISPR gene editing makes use of enzymes, particularly nucleases, that have been programmed to target specific sequences in the genome and then introduce edits. However, DNA cleavage and editing may occur at additional off-target sites in the genome that have similar but different DNA sequence from that of the intended site.
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