Gene editing fixes flaw linked to bad hearts

Embryo editing
This sequence of images shows the development of embryos after co-injection of a gene-correcting enzyme and sperm from a donor with a genetic mutation known to cause a type of heart disease known as hypertrophic cardiomyopathy. (OHSU Photo)

Researchers from Oregon Health and Science University provided the details today on an experiment with human embryos that demonstrated how gene editing can repair a genetic flaw linked to heart disease.

The outlines of the experiment were reported last week, but the OHSU team held off on providing the details until today’s publication of the study in the journal Nature.

The team, led by OHSU senior researcher Shoukhrat Mitalipov, took advantage of a technique known as CRISPR-Cas9, which uses RNA guide molecules and enzymes to make targeted cuts in the DNA molecules that contain the human genetic code. Revised code can then be inserted into the snipped DNA.

The Nature study revealed that the team was able to fix a genetic mutation that causes hypertrophic cardiomyopathy, a common disease that can cause sudden heart failure and death.

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By Alan Boyle

Mastermind of Cosmic Log, contributor to GeekWire and Universe Today, author of "The Case for Pluto: How a Little Planet Made a Big Difference," past president of the Council for the Advancement of Science Writing.

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