California stem cell agency may fund tests to edit genes in human embryos
A California agency funded by taxpayers is considering whether it should support scientific research aimed at genetically modifying human embryos — work so controversial that the federal government won’t pay for it.
The state’s stem cell institute is reviewing its ethics guidelines to determine whether they are strong enough to safely allow studies in which scientists would attempt to edit the genes of embryos.
Just this month, Britain became the first government to approve such an experiment, where a powerful new technology known as CRISPR will be used to edit genes in human embryos.
In the British research, the early-stage embryos will be studied and destroyed
after 14 days. The work is aimed at looking at the genes that are active just after fertilization.
It is illegal in Britain — but not in the United States — to use genetically modified embryos to start a pregnancy.
Kevin McCormack, a spokesman for the California Institute for Regenerative Medicine, said a committee is debating whether the institute should fund similar human gene-editing research using CRISPR.
The controversial technology, developed with the help of scientists at UC Berkeley, allows DNA to be edited with unparalleled precision. It has set off a biotech gold rush as scientists imagine its commercial uses and found start-up companies that are attracting hundreds of millions of dollars in venture capital.
The 3-year-old technology allows for the genomes of all living things to be spliced and revised. It has already been used to create wheat resistant to fungal diseases, correct genetic errors that cause muscular dystrophy in mice and engineer monkeys to have targeted mutations.
In medicine, scientists say it has the potential to help cure devastating hereditary disorders such as Huntington’s disease.
But it has also set off widespread alarm about the possibility that scientists could use it to genetically modify children and change the human race.
“This is not safe,” said Marcy Darnovsky at the Center for Genetics and Society in Berkeley. “It’s still way too early to try such an experiment on a human being.”
Last April, Francis Collins, director of the National Institutes of Health, said that the federal agency would not fund such research.
“Altering the human germ line in embryos for clinical purposes … has been viewed almost universally as a line that should not be crossed,” Collins explained in making the announcement.
In December, an international group of scientists called for what effectively would be a moratorium on making permanent changes to the human genome.
The group, convened by the National Academy of Sciences and scientists from China and Britain, said it would be “irresponsible to proceed” with such research until there was “broad societal consensus” on whether making inheritable changes to the human genome was appropriate.
The California Institute for Regenerative Medicine was created by voter approval of Proposition 71 in 2004. The agency is distributing
$3 billion from state taxpayers to stem cell researchers in the hope of discovering promising new cures for Parkinson’s, diabetes and a host of other diseases. The agency has about $900 million left to distribute.
The agency’s current regulations say that no money can be used to transfer a genetically modified human embryo to a woman to start a pregnancy. But some experts worry that agency-funded researchers could later turn to other sources to finance the reproductive stage of their work.
“If you have genetically modified embryos in labs around the state,” Darnovsky said, “what’s to stop them from being used to initiate a pregnancy?”
Many other questions were raised last week when the stem cell agency’s committee met in Los Angeles to review its policies on the editing of human genes.
“Do we need to think about the rights of the embryo donor?” asked board member Jeff Sheehy. “If they have a severe inheritable disease and the embryo they donated for research has been edited with CRISPR or other tools to remove that potential, do they have a right to know about that or even access to that technology for their own use?”
McCormack, the agency’s spokesman, said the committee will try to answer such questions and come back with recommendations that will go to the full board for consideration — a process expected to take a couple of months.
“So far we have not funded any research that involves CRISPR, nor have we received any proposals for funding using that technology,” McCormack said. “But that’s probably just a matter of time.”
Last year, Chinese scientists stunned the scientific community by revealing that they had already used CRISPR to genetically modify human embryos. To try to head off ethical concerns, the scientists had used embryos they said were non-viable and could not result in a live birth.
In their experiment, the researchers had attempted to modify a gene responsible for a potentially fatal blood disorder. But they experienced multiple problems, including finding a surprising number of off-target mutations in other parts of the genome.
The Chinese team said their results showed the serious barriers to using the gene-editing technique in humans.
Twitter: @melodypetersen
