In December 2015, a group of worldwide 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. They're said to have shown in experiments with "many tens" of human embryos that they can correct genetic mutations that cause disease while avoiding mosaicism and off-target effects.
The effort, led by Shoukhrat Mitalipov of Oregon Health and Science University in the U.S., involved changing the DNA of a large number of one-cell embryos with the controversial gene-editing technique CRISPR.
Scientists in China were the first to carry out gene editing on human embryos in 2015, although with mixed results, the British journal Nature reported.
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The goal is to be able to correct defective genes that would cause inherited diseases.
Many critics are anxious that the practice could lead to "designer babies" that are engineered with genetic enhancements.
A team of USA researchers for the first time ever edited human embryo DNA, the Massachusetts Institute of Technology (MIT) Tech Review revealed Wednesday.
But in February, a report from the U.S. National Academy of Sciences and the U.S. National Academy of Medicine said that clinical trials for gene editing of human reproductive cells "could be permitted in the future, but only for serious conditions under stringent oversight".
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Michio Hirano, an American neurology expert from Columbia Medical Center in NY who has designed the experimental treatment. Charlie, who is 11 months old, suffers from a rare genetic condition which causes progressive muscle weakness.
It involves technology called "CRISPR" to alter genes in embryos.
Now Mitalipov's team has apparently managed to reduce the rate of mosaicism by injecting the CRISPR machinery at an earlier stage, at the same time as the eggs were fertilised with sperm.
Mitalipov's results are still "pending publication", he told MIT. It's unclear what illnesses were involved exactly, but they used sperm donated by subjects with various inheritable diseases. Until the numbers are published, it will not be clear to what extent this reduced mosaicism. But Mitalipov's research, if it passes peer review, would be a significant step for American scientists.
"It is proof of principle that it can work", the researcher said.
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