“‘We feel it’s critical that the scientific community consider the potential hazards of all off-target mutations caused by CRISPR, including single nucleotide mutations and mutations in non-coding regions of the genome,’ says co-author Stephen Tsang, MD, PhD, the Laszlo T. Bito Associate Professor of Ophthalmology and associate professor of pathology and cell biology at Columbia University Medical Center and in Columbia’s Institute of Genomic Medicine and the Institute of Human Nutrition.”2
Because CRISPR is so fast and precise, scientists have been able to work quickly toward better understand of the role of genes in disease. And the tech has raised hopes that more powerful gene therapies than we currently have could delete or repair flawed genes,3 not just add new genes. In fact, the first clinical trial to deploy CRISPR is currently underway in China and a U.S. trial is set to begin in 2018.
However, researchers must still work on improving the CRISPR system to increase the efficiency of their editing:
“In the new study, the researchers sequenced the entire genome of mice that had undergone CRISPR gene editing in the team’s previous study and looked for all mutations, including those that only altered a single nucleotide.
The researchers determined that CRISPR had successfully corrected a gene that causes blindness, but Kellie Schaefer, a PhD student in the lab of Vinit Mahajan, MD, PhD, associate professor of ophthalmology at Stanford University, and co-author of the study, found that the genomes of two independent gene therapy recipients had sustained more than 1,500 single-nucleotide mutations and more than 100 larger deletions and insertions. None of these DNA mutations were predicted by computer algorithms that are widely used by researchers to look for off-target effects.”4
This means that researchers who aren’t using “whole genome sequencing to find off-target effects”5 might still be missing important mutations that have taken place. Taking extra care is going to be important. Like double and triple checking your math test.
Only way more important.
We will be watching this technology closely.
