Researchers in the US have developed a new CRISPR method for treating genetic conditions using nickases that they say is safer and more effective.
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University of California San Diego (UCSD)
How engineered RNA binding proteins delivered in vivo by gene therapy could treat myotonic dystrophy
In this article, Dr Jim Burns discusses promising pre-clinical results of how a new platform could treat the root cause of many devastating genetic diseases including myotonic dystrophy type 1.