Group of Scientists and Bioethicists Back Genetic Modification of Human Embryos

UK Approves Three-Person IVF Babies 23 comments

The BBC reports that three-person IVF will soon be legal in the United Kingdom. The procedure involves replacing mitochondrial DNA in an embryo from that of a second woman in order to eliminate deadly mitochondrial genetic disorders. Alana Saarinen was successfully conceived in the U.S. using the procedure back in 2000, but the FDA banned ooplasm transfer in 2001.

The UK has now become the first country to approve laws to allow the creation of babies from three people. The modified version of IVF has passed its final legislative obstacle after being approved by the House of Lords. The fertility regulator will now decide how to license the procedure to prevent babies inheriting deadly genetic diseases. The first baby could be born as early as 2016. A large majority of MPs in the House of Commons approved "three-person babies" earlier this month. The House of Lords tonight rejected an attempt to block the plan by a majority of 232. Estimates suggest 150 couples would be suitable to have babies through the technique each year.

Additional coverage at Wired UK and The Guardian.

Chinese Scientists Have Genetically Modified Human Embryos 27 comments

takyon writes:

A team of researchers led by Junjiu Huang at the Sun Yat-sen University in Guangzhou have reported human germline modification using CRISPR:

In a world first, Chinese scientists have reported editing the genomes of human embryos. The results are published in the online journal Protein & Cell and confirm widespread rumours that such experiments had been conducted — rumours that sparked a high-profile debate last month about the ethical implications of such work.

In the paper, researchers led by Junjiu Huang, a gene-function researcher at Sun Yat-sen University in Guangzhou, tried to head off such concerns by using 'non-viable' embryos, which cannot result in a live birth, that were obtained from local fertility clinics. The team attempted to modify the gene responsible for β-thalassaemia, a potentially fatal blood disorder, using a gene-editing technique known as CRISPR/Cas9. The researchers say that their results reveal serious obstacles to using the method in medical applications.

[...] A Chinese source familiar with developments in the field said that at least four groups in China are pursuing gene editing in human embryos.

While some embryos were successfully edited, the use of CRISPR/Cas9 was not nearly as reliable as desired:

The team injected 86 embryos and then waited 48 hours, enough time for the CRISPR/Cas9 system and the molecules that replace the missing DNA to act — and for the embryos to grow to about eight cells each. Of the 71 embryos that survived, 54 were genetically tested. This revealed that just 28 were successfully spliced, and that only a fraction of those contained the replacement genetic material. "If you want to do it in normal embryos, you need to be close to 100%," Huang says. "That's why we stopped. We still think it's too immature."

NIH Won't Fund Human Germline Modification 21 comments

takyon writes:

A week after a Chinese team reported semi-successful modification of human embryos, Dr. Francis Collins, director of the National Institutes of Health, has said in a statement that his agency will not fund any research involving human germline modification:

The concept of altering the human germline in embryos for clinical purposes has been debated over many years from many different perspectives, and has been viewed almost universally as a line that should not be crossed. Advances in technology have given us an elegant new way of carrying out genome editing, but the strong arguments against engaging in this activity remain. These include the serious and unquantifiable safety issues, ethical issues presented by altering the germline in a way that affects the next generation without their consent, and a current lack of compelling medical applications justifying the use of CRISPR/Cas9 in embryos.

Practically, there are multiple existing legislative and regulatory prohibitions against this kind of work. The Dickey-Wicker amendment prohibits the use of appropriated funds for the creation of human embryos for research purposes or for research in which human embryos are destroyed (H.R. 2880, Sec. 128). Furthermore, the NIH Guidelines state that the Recombinant DNA Advisory Committee, "...will not at present entertain proposals for germ line alteration". It is also important to note the role of the U.S. Food and Drug Administration (FDA) in this arena, which applies not only to federally funded research, but to any research in the U.S. The Public Health Service Act and the Federal Food, Drug, and Cosmetic Act give the FDA the authority to regulate cell and gene therapy products as biological products and/or drugs, which would include oversight of human germline modification. During development, biological products may be used in humans only if an investigational new drug application is in effect (21 CFR Part 312).

However, some scientists aren't joining the chorus of "universal" criticism:

George Church, a geneticist at Harvard Medical School in Boston, Massachusetts, disagrees that the technology is so immature. He says that the researchers did not use the most up-to-date CRISPR/Cas9 methods and that many of the researchers' problems could have been avoided or lessened if they had.

Although researchers agree that a moratorium on clinical applications is needed while the ethical and safety concerns of human-embryo editing are worked out, many see no problem with the type of research that Huang's team did, in part because the embryos could not have led to a live birth. "It's no worse than what happens in IVF all the time, which is that non-viable embryos are discarded," says John Harris, a bioethicist at the University of Manchester, UK. "I don't see any justification for a moratorium on research," he adds. Church, meanwhile, notes that many of the earliest experiments with CRISPR/Cas9 were developed in human induced pluripotent stem cells, adult cells that have been reprogrammed to have the ability to turn into any cell type, including sperm and eggs. He questions whether Huang's experiments are any more intrinsically problematic.

The Rapid Rise of CRISPR 7 comments

takyon writes:

Nature has a comprehensive analysis and history of CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats), the disruptive technique that is allowing genetic engineering and gene therapy to flourish:

CRISPR methodology is quickly eclipsing zinc finger nucleases and other [genetic] editing tools (see 'The rise of CRISPR'). For some, that means abandoning techniques they had taken years to perfect. "I'm depressed," says Bill Skarnes, a geneticist at the Wellcome Trust Sanger Institute in Hinxton, UK, "but I'm also excited." Skarnes had spent much of his career using a technology introduced in the mid-1980s: inserting DNA into embryonic stem cells and then using those cells to generate genetically modified mice. The technique became a laboratory workhorse, but it was also time-consuming and costly. CRISPR takes a fraction of the time, and Skarnes adopted the technique two years ago.
Researchers have traditionally relied heavily on model organisms such as mice and fruit flies, partly because they were the only species that came with a good tool kit for genetic manipulation. Now CRISPR is making it possible to edit genes in many more organisms. In April, for example, researchers at the Whitehead Institute for Biomedical Research in Cambridge, Massachusetts, reported using CRISPR to study Candida albicans, a fungus that is particularly deadly in people with weakened immune systems, but had been difficult to genetically manipulate in the lab. Jennifer Doudna, a CRISPR pioneer at the University of California, Berkeley, is keeping a list of CRISPR-altered creatures. So far, she has three dozen entries, including disease-causing parasites called trypanosomes and yeasts used to make biofuels.
Yet the rapid progress has its drawbacks. "People just don't have the time to characterize some of the very basic parameters of the system," says Bo Huang, a biophysicist at the University of California, San Francisco. "There is a mentality that as long as it works, we don't have to understand how or why it works." That means that researchers occasionally run up against glitches. Huang and his lab struggled for two months to adapt CRISPR for use in imaging studies. He suspects that the delay would have been shorter had more been known about how to optimize the design of guide RNAs, a basic but important nuance.

Original Submission

UK Scientist Makes the Case for Editing Human Embryos 20 comments

takyon writes:

Dr. Kathy Niakan from the Francis Crick Institute is seeking approval from the UK's Human Fertilisation and Embryology Authority in order to genetically modify human embryos:

A scientist has been making her case to be the first in the UK to be allowed to genetically modify human embryos. Dr Kathy Niakan said the experiments would provide a deeper understanding of the earliest moments of human life and could reduce miscarriages. The regulator, the Human Fertilisation and Embryology Authority (HFEA), will consider her application on Thursday. If Dr Niakan is given approval then the first such embryos could be created by the summer.
[...] Dr Niakan, from the Francis Crick Institute, said: "We would really like to understand the genes needed for a human embryo to develop successfully into a healthy baby. The reason why it is so important is because miscarriages and infertility are extremely common, but they're not very well understood."
Of 100 fertilised eggs, fewer than 50 reach the blastocyst stage, 25 implant into the womb and only 13 develop beyond three months. She says that understanding what is supposed to happen and what can go wrong could improve IVF. "We believe that this research could really lead to improvements in infertility treatment and ultimately provide us with a deeper understanding of the earliest stages of human life."
However, she says the only way to do this is to edit human embryos. Many of the genes which become active in the week after fertilisation are unique to humans, so they cannot be studied in animal experiments. "The only way we can understand human biology at this early stage is by further studying human embryos directly," Dr Niakan said. Her intention is to use one of the most exciting recent scientific breakthroughs - Crispr gene editing - to turn off genes at the single-cell stage and see what happens. [...] She aims to start with the gene Oct4 which appears to have a crucial role.

Original Submission

UK Regulator Approves Gene Editing in Human Embryos 12 comments

takyon writes:

Scientists with The Francis Crick Institute have received UK Human Fertilisation and Embryology Authority (HFEA) approval for using gene editing techniques on human embryos:

The aim of the research, led by Dr Kathy Niakan, a group leader at the Crick, is to understand the genes human embryos need to develop successfully. The work carried out at the Crick will be for research purposes and will look at the first seven days of a fertilised egg's development (from a single cell to around 250 cells).
[...] In line with HFEA regulations, any donated embryos will be used for research purposes only and cannot be used in treatment. These embryos will be donated by patients who have given their informed consent to the donation of embryos which are surplus to their IVF treatment. The genome editing research now needs to gain ethical approval and, subject to that approval, the research programme will begin within the next few months.

The decision marks the first approval by a regulator of gene editing in human embryos:

In a world-first last year, scientists in China announced they had carried out gene editing in human embryos to correct a gene that causes a blood disorder. Prof Robin Lovell-Badge, a scientific advisor to the UK's fertility regulator, told the BBC: "China has guidelines, but it is often unclear exactly what they are until you've done it and stepped over an unclear boundary. This is the first time it has gone through a properly[sic] regulatory system and been approved."

[Continues...]

Francis Collins Retains Position as Director of the National Institutes of Health 6 comments

takyon writes:

Francis Collins will remain the director of the National Institutes of Health, for now:

Ending weeks of speculation, President-elect Donald Trump has asked National Institutes of Health (NIH) Director Francis Collins to remain in his position. It is not clear for how long. "We just learned that Dr. Collins has been held over by the Trump administration," an NIH spokesperson said in a statement. "We have no additional details at this time."
Collins, a geneticist who has headed the $32 billion NIH for the past 8 years, has been campaigning to keep his job and met with Trump last week. On Wednesday, he told a reporter at the World Economic Forum in Davos, Switzerland, that he still didn't know what his fate would be. But although Collins had the support of key Republicans in Congress, he has been one of several candidates for the NIH post, including Representative Andy Harris (R–MD).

Original Submission

First Known Attempt at Genetically Modifying Human Embryos in the U.S. is an Apparent Success 28 comments

takyon writes:

U.S. scientists have genetically modified human embyros using CRISPR and have apparently avoided the worst of the off-target effects that have plagued previous efforts. The results are unpublished and the team is not commenting yet:

The first known attempt at creating genetically modified human embryos in the United States has been carried out by a team of researchers in Portland, Oregon, Technology Review has learned.
The effort, led by Shoukhrat Mitalipov of Oregon Health and Science University, involved changing the DNA of a large number of one-cell embryos with the gene-editing technique CRISPR, according to people familiar with the scientific results.
Until now, American scientists have watched with a combination of awe, envy, and some alarm as scientists elsewhere were first to explore the controversial practice. To date, three previous reports of editing human embryos were all published by scientists in China.
Now 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.
Although none of the embryos were allowed to develop for more than a few days—and there was never any intention of implanting them into a womb—the experiments are a milestone on what may prove to be an inevitable journey toward the birth of the first genetically modified humans.

Also at STAT News.

Previously: Chinese Scientists Have Genetically Modified Human Embryos
NIH Won't Fund Human Germline Modification
Group of Scientists and Bioethicists Back Genetic Modification of Human Embryos
The International Summit on Human Gene Editing
UK Scientist Makes the Case for Editing Human Embryos
Second Chinese Team Reports Gene Editing in Human Embryos
Scientists Keep Human Embryos Alive Longer Outside of the Womb
Francis Collins Retains Position as Director of the National Institutes of Health

Original Submission

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