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No Cut Gene Editing

December 11, 2017

A new form of gene therapy could correct genetic disorders with less risk.



No-cut gene-editing. I’m Bob Hirshon and this is Science Update.

Scientists have a powerful new tool called CRISPR-cas9 that could let them treat genetic disorders by cutting cut out bad segments of DNA and pasting in corrected ones. But cutting DNA can pose risks, accidentally introducing new, unwanted gene mutations. In the journal Cell, Salk Institute researcher Juan Carlos Belmonte and his colleagues report on a new way to use CRISPR-cas9 to insert corrective genes into a cell, overriding the disease gene, instead of removing it.


So the idea here is trying to do the genetic editing but without cutting the DNA.


The researchers have used the method to treat mouse models of kidney disease, diabetes and muscular dystrophy. But Belmonte says theyll need additional lab studies before moving to human trials.  I’m Bob Hirshon, for AAAS, the science society.