A Single Dose of CRISPR Treats a Progressive Genetic Disease in Humans

The preliminary data suggests that this could be a promising new treatment.

“CRISPR Cas9” by National Institutes of Health (NIH) is marked with CC PDM 1.0

A rare genetic disease causes the buildup of misshaped proteins in the heart and nerve cells. Scientists injected molecular scissors, to “cut out” the genetic mutation that caused it. In a preliminary human trial, this…