Embryo gene-editing to eliminate genetic disease

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  • #72123
    nittany ram
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    Link: http://www.independent.co.uk/news/health/human-embryo-editing-crispr-9-baby-treatment-advance-paediatrics-designer-babies-a7873746.html
    aily Edition

    NewsHealth
    Human embryo editing breakthrough is a ‘major advance’ towards controversial treatments for babies
    The treatment could help rid babies of genetic diseases. But the ethical and legal considerations need urgent work, experts have warned

    Andrew Griffin @_andrew_griffin 7 days ago94 comments

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    The Independent Online
    istock-475618532.jpg
    Picture: Getty/iStockphoto
    A landmark study suggests that scientists could soon edit out genetic mutations to prevent babies being born with diseases. The technique could eventually let doctors remove inherited conditions from embryos before they go on to become a child.

    That, in turn, opens the possibility for inherited diseases to be wiped out entirely, according to doctors. But experts have warned that urgent work is needed to answer the ethical and legal questions surrounding the work.

    Though the scientists only edited out mutations that could cause diseases, it modified the nuclear DNA that sits right at the heart of the cell, which also influences personal characteristics such as intelligence, height, facial appearance and eye colour.

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    The breakthrough means that “the possibility of germline genome editing has moved from future fantasy to the world of possibility, and the debate about its use, outside of fears about the safety of the technology, needs to run to catch up”, said Professor Peter Braude from King’s College London. Scientists warned that soon the public could demand such treatment – and that the world might not be ready.

    “Families with genetic diseases have a strong drive to find cures,” said Yalda Jamshidi, reader in genomic medicine at St George’s, University of London. “Whilst we are just beginning to understand the complexity of genetic disease, gene-editing will likely become acceptable when its potential benefits, both to individuals and to the broader society, exceeds its risks.”

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    The new research, published in Nature, marks the first time the powerful Crispr-Cas9 tool has been used to fix mutations. The US study destroyed the embryos after just a few days and the work remains at an experimental stage.

    In the study, scientists fertilised donor eggs with sperm that included a gene that causes a type of heart failure. As the eggs were fertilised, they also applied the gene-editing tool, which works like a pair of specific scissors and cuts away the defective parts of the gene.

    When those problematic parts are cut away, the cells can repair themselves with the healthy versions and so get rid of the mutation that causes the disease. Some 42 out of 58 embryos were fixed so that they didn’t carry the mutation – stopping a disease that usually has a 50 per cent chance of being passed on.

    If those embryos had been allowed to develop into children, then they would no longer have carried the disease. That would stop them from being vulnerable to hypertrophic cardiomyopathy – and would save their children, too.

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    Gene editing technique named scientific breakthrough of the year
    “Every generation on would carry this repair because we’ve removed the disease-causing gene variant from that family’s lineage,” said Dr Shoukhrat Mitalipov, from Oregon Health and Science University, who led the study.

    “By using this technique, it’s possible to reduce the burden of this inheritable disease on the family and eventually the human population.”

    The heart problem is just one of more than 10,000 conditions that are caused by an error in the gene. The same tool could be used to cut out those faults for all of those, and eventually could be used to target cancer mutations.

    The work could lead to treatments that would be given to patients, once it becomes more efficient and safe. Using such a treatment on humans is illegal in both the US and the UK – but some experts expect that law will soon be changed, and that the legal and ethical frameworks need to catch up with the technology.

    There is some suggestion that the editing work could take place in the UK. Though using the research as treatment is illegal there as well as the US, the regulatory barriers are much higher in America and look unlikely to be changed.

    In the US, there are various regulations and restrictions on how embryos can be edited, including stipulations that such work can’t be carried out with taxpayers’ money. UK regulators are more relaxed and liberal about those restrictions, leading to suggestions that it could eventually become the home of such work in the west.

    The UK has become the first country that allows mitochondrial replacement therapy, another treatment that opponents warn could allow for the creation of designer babies.

    embryo-dna-do-not-reuse-pa.jpg
    Individual cells days after injection (PA)
    “UK researchers can apply for a licence to edit human embryos in research, but offering it as a treatment is currently illegal,” said a spokesperson for the Human Fertilisation and Embryology Authority (HEFA), which would regulate any such experiments.

    “Introducing new, controversial techniques is not just about developing the science – gene editing would need to offer new options to couples at risk of having a child with a genetic disease, beyond current treatments like embryo testing.

    “Our experience of introducing mitochondrial donation in the UK shows that high-quality public discussion about the ethics of new treatments, expert scientific advice and a robust regulatory system are crucial when considering new treatments of this kind.”

    Doctors said that any change in the law would have to strictly keep such treatment to being used for medical reasons, and not for “designer babies” that have other characteristics edited out.

    “It may be that some countries never permit germline genome editing because of moral and ethical concerns,” said Professor Joyce Harper from University College London. “If the law in the UK was changed to allow genome editing, it would be highly regulated by the Human Fertilisation and Embryology Authority, as is PGD, to ensure it is only used for medical reasons.”

    But that work has already received significant opposition.

    Dr David King, director of the Human Genetics Alert, which opposes all tampering with the human genome, said: “If irresponsible scientists are not stopped, the world may soon be presented with a fait accompli of the first GM baby.

    “We call on governments and international organisations to wake up and pass an immediate global ban on creating cloned or GM babies, before it is too late.”

    Professor Robin Lovell-Badge from the Francis Crick Institute said the research only appears to work when the father is carrying the defective gene, and that it would not work for more sophisticated alterations. “The possibility of producing designer babies, which is unjustified in any case, is now even further away,” he said.

    More about: Crisprgene editing

    #72124
    nittany ram
    Moderator

    Designer babies highly unlikely….

    Link: https://www.nytimes.com/2017/08/04/science/gene-editing-embryos-designer-babies.html

    Gene Editing for ‘Designer Babies’?
    Highly Unlikely, Scientists Say
    Fears that embryo modification could allow parents to custom
    order a baby with Lin-Manuel Miranda’s imagination or
    Usain Bolt’s speed are closer to science fiction than science.

    By PAM BELLUCKAUG. 4, 2017

    Now that science is a big step closer to being able to fiddle with the genes of a human embryo, is it time to panic? Could embryo editing spiral out of control, allowing parents to custom-order a baby with Lin-Manuel Miranda’s imagination or Usain Bolt’s speed?

    News that an international team of scientists in Oregon had successfully modified the DNA of human embryos has renewed apprehensions that babies will one day be “designed.” But there are good reasons to think that these fears are closer to science fiction than they are to science.

    Here is what the researchers did: repair a single gene mutation on a single gene, a defect known to cause — by its lonesome — a serious, sometimes fatal, heart disease.

    Here is what science is highly unlikely to be able to do: genetically predestine a child’s Ivy League acceptance letter, front-load a kid with Stephen Colbert’s one-liners, or bake Beyonce’s vocal range into a baby.

    That’s because none of those talents arise from a single gene mutation, or even from an easily identifiable number of genes. Most human traits are nowhere near that simple.

    “Right now, we know nothing about genetic enhancement,” said Hank Greely, director of the Center for Law and the Biosciences at Stanford. “We’re never going to be able to say, honestly, ‘This embryo looks like a 1550 on the two-part SAT.’”

    Even with an apparently straightforward physical characteristic like height, genetic manipulation would be a tall order. Some scientists estimate height is influenced by as many as 93,000 genetic variations. A recent study identified 697 of them.

    Can Gene Editing Actually Do That?
    A new technique known as Crispr has revolutionized humans’ ability to edit DNA. See if you can identify whether a given development has already happened, could eventually happen or is pure fiction.

    “You might be able to do it with something like eye color,” said Robin Lovell-Badge, a professor of genetics and embryology at the Francis Crick Institute in London.

    But “if people are worried about designer babies, they’re normally thinking of doing special — different things than the normal genetic stuff.”

    The gene-modification process used in the new study also turns out to be somewhat restrictive. After researchers snipped the harmful mutation from the male gene, it copied the healthy sequence from that spot on the female gene.

    That was a surprise to the scientists, who had inserted a DNA template into the embryo, expecting the gene to copy that sequence into the snipped spot, as occurs with gene editing in other body cells. But the embryonic genome ignored that template, suggesting that to repair a mutation on one parent’s gene in an embryo, a healthy DNA sequence from the other parent is required.

    “If you can’t introduce a template, then you can’t do anything wild,” Dr. Lovell-Badge said. “This doesn’t really help you make designer babies.”

    Talents and traits aren’t the only thing that are genetically complex. So are most physical diseases and psychiatric disorders. The genetic message is not carried in a 140-character tweet — it resembles a shelf full of books with chapters, subsections and footnotes.

    So embryonic editing is unlikely to prevent most medical problems.

    But about 10,000 medical conditions are linked to specific mutations, including Huntington’s disease, cancers caused by BRCA genes, Tay-Sachs disease, cystic fibrosis, sickle cell anemia, and some cases of early-onset Alzheimer’s. Repairing the responsible mutations in theory could eradicate these diseases from the so-called germline, the genetic material passed from one generation to the next. No future family members would inherit them.

    A composite image showing the development of embryos after injection of a gene-correcting enzyme and sperm from a donor with a genetic mutation known to cause hypertrophic cardiomyopathy. Credit Oregon Health & Science University
    But testing editing approaches on each mutation will require scientists to find the right genetic signpost, often an RNA molecule, to guide the gene-snipping tool.

    In the study reported this week, it took 10 tries to find the right RNA, said Juan Carlos Izpisua Belmonte, a co-author and geneticist at the Salk Institute.

    Dr. Greely noted that while scientists work to get human embryonic editing ready for clinical trials (currently illegal in the United States and many countries), alternate medical treatments for these diseases might be developed. They may be simpler and cheaper.

    “How good one technique is depends on how good the alternatives are, and there may be alternatives,” he said.

    • This reply was modified 6 years, 8 months ago by nittany ram.
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