In US first, scientists edit genes of human embryos

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In a step that some of the nation's leading scientists have long warned against and that has never before been accomplished, biologists in OR have edited the DNA of viable human embryos efficiently and apparently with few mistakes, according to a report in Technology Review.

In a first in the United States, scientists using the "CRISPR" genome-editing technique, have successfully corrected the DNA in human embryos that carried inherited diseases.

The first experiment using CRISPR to alter the DNA of human embryos, in 2015, used embryos obtained from fertility clinics that had such serious genetic defects they could never have developed.

Those who oppose the work worry that it could lead to an era of "designer babies", modified to fit a preconceived ideal, while supporters suggest it's a miraculous discovery that could one day eliminate many childhood and lifelong diseases.

According to OHSU spokesperson Eric Robinson, the result of the peer-reviewed study are expected to be published soon in a scientific journal.

CRISPR, a revolutionary gene-editing technique, has opened up enormous potential to battle diseases and genetic faults. The technology works as a kind of scissors that can snip selected, unwanted parts of the genome, replacing these with new DNA.

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Worldwide experts in the emerging field of gene therapy said it would be hard to assess the results until the OR team fully published its findings. "Mitalipov is believed to have broken new ground both in the number of embryos experimented upon and by demonstrating that it is possible to safely and efficiently correct defective genes that cause inherited diseases". They said although basic and preclinical research should be allowed, edited human embryos should not be used to establish a pregnancy.

In December 2015, a group of global scientists and ethicists, including some from China, assembled by the US National Academy of Sciences said it would be irresponsible to use DNA editing tools to alter the genomes of human embryos, eggs, or sperm until safety, ethical and legal issues were resolved.

CRISPR-Cas9 is a tool for making precise edits in DNA, discovered in bacteria. The only previous work like this has been reported in China.

'I don't think it's the start of clinical trials yet, but it does take it further than anyone has before'. But Mitalipov was able to "significantly" reduce mosaicism, according to MIT Technology Review.

In this way, researchers can precisely turn off specific genes in the genome.

The need for it is clear, he added: "Our research has suggested that there are far more disease-associated mutations in the general public than was previously suspected".