“CRISPR babies” scientist He Jiankui tried to publish a paper describing additional experiments that made heritable changes in the DNA of human embryos, much like those that led to the birth of the world’s first genome-edited humans. But the paper was rejected by an international journal after outside scientists raised concerns about both its ethics and its scientific validity, STAT has learned.
The rejected paper did not report starting a pregnancy with the edited embryos, and “there is no plan” to do so, said Ryan Ferrell, He’s public relations consultant. It is nevertheless one of a tiny handful of experiments editing normal human embryos: A Chinese lab did it in 2017, a U.S. lab followed a few months later, and another Chinese lab did it this summer. And it would make He’s one of only two labs in the world known to have edited human embryos to alter genes for more than one disease.
In the so-far unpublished, 55-page paper, He and his colleagues report used CRISPR-Cas9 to target the gene PCSK9 in very early embryos of mice, monkeys, and people. When both of an individual’s PCSK9 genes (one inherited from mom, the other from dad) have a mutation that increases the activity of the protein the gene makes, the result is sky-high cholesterol; it is this inherited, “familial hypercholesterolemia” that He and his colleagues wrote they were trying to prevent.
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