Although not ready yet, powerful gene editing tools may one day be used on human embryos, eggs and sperm to remove genes that cause inherited diseases, according to a report by US scientists and ethicists released on Tuesday.
The report (paywall) from the National Academy of Sciences (NAS) and the National Academy of Medicine (paywall) said scientific advances make gene editing in human reproductive cells “a realistic possibility that deserves serious consideration.”
The statement signals a softening in approach over the use of the technology known as CRISPR-Cas9, which has opened up new frontiers in genetic medicine because of its ability to modify genes quickly and efficiently.
In December 2015, scientists and ethicists at an international meeting held at the NAS in Washington said it would be “irresponsible” to use gene editing technology in human embryos for therapeutic purposes, such as to correct genetic diseases, until safety and efficacy issues are resolved.
The latest NAS report now says clinical trials for human genome editing could be permitted, “but only for serious conditions under stringent oversight.”
CRISPR-Cas9 works as a type of molecular scissors that can selectively trim away unwanted parts of the genome and replace them with new stretches of DNA.
Genome editing is already being planned for use in clinical trials on people to correct diseases caused by a single gene mutation, such as sickle cell disease. But these therapies affect only the patient.
The concern is over the use of the technology in human reproductive cells or early embryos because the changes would be passed along to offspring.
Research using the powerful technique is plowing ahead even as researchers from the University of California and the Broad Institute battle for control over the CRISPR patent.
Although gene editing of human reproductive cells to correct inherited diseases “must be approached with caution, caution does not mean prohibition,” the committee said in a statement.