The CRISPR–Cas9 enzyme complex (blue and grey) edits DNA (purple). Participants in one of the earliest clinical trials of a CRISPR-based therapy experienced no serious side effects. Credit: Ella Maru Studio/Science Photo Library
Preliminary results from one of the earliest clinical trials of
CRISPR—Cas9 provide evidence that the technique is safe and feasible to use for treating human diseases.
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Nature 578, 195 (2020)
doi: https://doi.org/10.1038/d41586-020-00339-3
Science (2020)
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CRISPR (clustered regularly interspaced short palindromic repeats) is a family of DNA sequences found in the genomes of prokaryotic organisms such as bacteria and archaea. These sequences are derived from DNA fragments of bacteriophages that had previously infected the prokaryote. They are used to
detect and destroy DNA from similar bacteriophages during subsequent infections. CRISPR technology is a simple yet powerful tool for editing genomes. It allows researchers to easily alter DNA sequences and modify gene function. Its many potential applications include correcting genetic defects, treating and preventing the spread of diseases and improving crops.