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August 1, 2017

CRISPR-Based System Uncovers Key Regulators of Disease-Related Genes

JAMA. 2017;318(5):412-413. doi:10.1001/jama.2017.9216

Using CRISPR/Cas9 gene editing technology, investigators have developed a method that may reveal the functions of regulatory elements in the human genome that do not code for proteins but have critical roles in health and disease.

Adapted from a bacterial host-defense system, CRISPR (clustered, regularly interspaced, palindromic repeats) uses the DNA-cutting enzyme Cas9 bound to a short RNA guide strand that can be tailored to home to a specific genetic sequence. In a recent Nature Biotechnologystudy, researchers at Duke University used a modified version of this system to target DNA sequences that don’t encode genes but rather dictate if and when particular genes will be expressed.