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'Jaw-dropping' breakthrough: CRISPR technique heralds genetic revolution

Discussion in 'Other Health News and Research' started by Waverunner, Nov 12, 2013.

  1. Waverunner

    Waverunner Senior Member

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    This form of therapy could not only revolutionize the treatment of hereditary diseases but could also include the cure for HIV and other diseases including CFS.

    http://www.independent.co.uk/news/s...nique-heralds-genetic-revolution-8925295.html

    A breakthrough in genetics – described as “jaw-dropping” by one Nobel scientist – has created intense excitement among DNA experts around the world who believe the discovery will transform their ability to edit the genomes of all living organisms, including humans.

    The development has been hailed as a milestone in medical science because it promises to revolutionise the study and treatment of a range of diseases, from cancer and incurable viruses to inherited genetic disorders such as sickle-cell anaemia and Down syndrome.

    For the first time, scientists are able to engineer any part of the human genome with extreme precision using a revolutionary new technique called Crispr, which has been likened to editing the individual letters on any chosen page of an encyclopedia without creating spelling mistakes. The landmark development means it is now possible to make the most accurate and detailed alterations to any specific position on the DNA of the 23 pairs of human chromosomes without introducing unintended mutations or flaws, scientists said.

    The technique is so accurate that scientists believe it will soon be used in gene-therapy trials on humans to treat incurable viruses such as HIV or currently untreatable genetic disorders such as Huntington’s disease. It might also be used controversially to correct gene defects in human IVF embryos, scientists said.

    Until now, gene therapy has had largely to rely on highly inaccurate methods of editing the genome, often involving modified viruses that insert DNA at random into the genome – considered too risky for many patients.

    The new method, however, transforms genetic engineering because it is simple and easy to edit any desired part of the DNA molecule, right down to the individual chemical building-blocks or nucleotides that make up the genetic alphabet, researchers said.

    “Crispr is absolutely huge. It’s incredibly powerful and it has many applications, from agriculture to potential gene therapy in humans,” said Craig Mello of the University of Massachusetts Medical School, who shared the 2006 Nobel Prize for medicine for a previous genetic discovery called RNA interference.

    “This is really a triumph of basic science and in many ways it’s better than RNA interference. It’s a tremendous breakthrough with huge implications for molecular genetics. It’s a real game-changer,” Professor Mello toldThe Independent.

    “It’s one of those things that you have to see to believe. I read the scientific papers like everyone else but when I saw it working in my own lab, my jaw dropped. A total novice in my lab got it to work,” Professor Mello said.

    In addition to engineering the genes of plants and animals, which could accelerate the development of GM crops and livestock, the Crispr technique dramatically “lowers the threshold” for carrying out “germline” gene therapy on human IVF embryos, Professor Mello added...
  2. Firestormm

    Firestormm Senior Member

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    All a bit scary though don't you think? Genetic engineering. It's science fiction become fact. We'll be able to choose how we want our kids to look soon. Still, if it can help with chronic disease treatment, and/or fix genetic disease - all to the good. Thanks Waverunner.
  3. maryb

    maryb iherb code TAK122

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    thanks @Waverunner
    somehow I think the GM crops people will benefit before those of us with ME, but fantastic technology all the same.
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  4. anciendaze

    anciendaze Senior Member

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    Viruses have been engineering changes in our genomes for a very long time. Retroviruses actually insert their genes in chromosomes, and sometimes pick up genes from other viruses in the process. DNA viruses like those in the herpes group put their genes in separate bodies inside the nuclear membrane called episomes. These use the rest of the cell's biochemical machinery to reproduce proteins vital for their propagation. RNA viruses simply insert their own sequences at an intermediate stage like mRNA transcribed from our own genes. The end result is also the reproduction of the virus rather than any function vital to the cell.

    The debris from this long-term process is all over the genome. The precise percentage of the genome is debatable, (when do you call a sequence viral, and when a retrotransposon?) but the consequences are enormous. It's about time we found some way of editing genes short of selecting genes by selecting entire individuals. In the normal course of events deleting a defective gene takes place by a process called death.
  5. Waverunner

    Waverunner Senior Member

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    True, it looks like science fiction. What will life look like in 50 or 100 years? Will we be able to cure most diseases by then?

    Do you know what my only wish for us PWCs and all other people with chronic conditions would be? It's not to become a super human. I don't want to have super strong muscles, I don't want to be immune against viruses, I don't want to be able to hold my breath for 30 minutes without passing out. The only thing I want is to be healthy again. I want this fatigue to be gone, I want this brain fog to be gone, I want all these symptoms, that came along with this illness or developed as a consequence of it it to disappear. I want to leave this house and be able to live my own life again, a life, that is not determined by disease. I don't need wealth, I don't need an expensive car but what I do need is health. And if the theory, that PWCs have certain genetic mutations, which make them prone for this disease, is true, I only want one thing: Replace the mutated genes and fix the cause.

    Nature already knows how these healthy genes look like. All the healthy people carry them in their genome. I don't want scientists to design new genes, I only want them to find the healthy genes and insert them into my genome. I don't want them to develop new drugs, which only suppress symptoms, have to be taken for the rest of the life, cost billions of dollars and take 10-15 years till they reach the market. I want them to use CRISPR and replace faulty genes, so that every PWC and every person with a severe chronic condition can live life as it was intended to be. That's all I want.
  6. Overstressed

    Overstressed Senior Member

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    This cuts through the heart, because it's so sincere. I wish that for you, and for every individual suffering.

    Best wishes,
    OS.
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  7. alex3619

    alex3619 Senior Member

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    The biggest problem with genetic engineering is not fixing the genes. Its fixing them in all or most of a persons cells simultaneously. That is why plants are easier to fix - you don't fix the current plant, you fix bits of it, grow seeds, and test to see if those seeds contain the desired genes. Alternatively you use gene insertion into a cell, or fix that cell, then grow a new plant.

    There is also a question of long term genetic stability.

    Having said that, this does have enormous potential, but first we will not see that in human patients, but in embryos of people with genetic diseases. Human trials will be many many years off, and trials on human adults with disease will be even further off.

    We also have the issue that we don't yet know what genes to correct.
  8. Waverunner

    Waverunner Senior Member

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    The penetrance rate is important but doesn't have to be. In many cases it should be enough to only target certain tissue. Many people don't know this but we already have an approved treatment in Europe, that is based on gene therapy. It is called Glybera (http://www.uniqure.com/products/glybera/) and as far as I understood it, the patients only received one injection: "A single dose administration of Glybera resulted in a long-term presence and biological activity of the protein in the injected muscle."

    What you say, is completely right of course. The medical field and regulators weren't very fast during the last decades and probably won't be during the next decade. Gene therapy will be used in monogenic diseases first and it will take at least 10-20 years, till they get it going in other diseases as well.

    However, there are tens of thousands of desperate patients who pay huge amounts of money for clinics abroad, which offer stem cell treatment and all kinds of other things that mostly don't work, in order to cure their often fatal diseases. In most cases it is a pure rip-off. But now imagine this--> What if these clinics would actually offer something that works? What if they offer CRISPR gene editing for genetic diseases? If it worked, demand would be huge. You couldn't stop this in the US nor in Europe. Just compare it to the war on drugs, it fails on all fronts. If people want something and pay the right price, they get it.

    In the case of CRISPR it would be highly unethical to prevent people from accessing a working treatment but even if everything fails, people could still fly to countries that offer it. So the big concern shouldn't be if regulators approve CRISPR within the next 20 years or not, the big question is if it works and if we find the faulty genes which we need to replace.
    Last edited: Nov 13, 2013
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  9. Firestormm

    Firestormm Senior Member

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    @Waverunner can I ask what is probably a daft question? Thanks :)

    How do you determine a gentically healthy sequence with which to compare/take from for gene therapy?

    Actually, I have another:

    Would we not have to discover a biomarker - in genes - for ME before considering gene therapy for ME necessitating a large study and is anyone currently working on such a search?

    :)
    aimossy likes this.
  10. Waverunner

    Waverunner Senior Member

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    Very good questions. Since I'm a true layman, I have problems answering them but I will give it a try. Determining a healthy sequence should work by looking at healthy people, who don't have the disease. So take 1,000 CFS patients for example, sequence their genomes and compare the results to reference genomes. Despite the fact, that we are still at the beginning of the genome era, we have a good understanding of what genes play an important role for which disease. Take BRCA 1&2 as example. Despite the fact, that we already identified over a thousand different mutations (and we don't have a good understanding of what impact missense mutations have) in these two genes, we could identify their importance for breast cancer.

    And regarding your second question, you are right, we would need to identify the genetic culprits first, before we can replace the faulty genes. Are studies underway? Yes: http://phoenixrising.me/archives/18222

    "To have any hope of identifying many different diseases (or causative pathways) within the umbrella definition, a lot of patients are needed; and there are early plans for a study involving a 5,000-strong cohort of patients. The idea is to explore everything: phenotypes, genotypes, gene expression, cells, cytokines, metabolites and more."

    But even if everything fails, I can still give you hope. It will become cheaper and easier to analyse genomes. Sooner or later patients will be able to analyse their genomes alone. In the case of Nickolas Volker they only used his genome and figured out the cause for his disease (http://www.forbes.com/sites/matthewherper/2011/01/05/the-first-child-saved-by-dna-sequencing/). So we have to see where things go from here.
    Overstressed likes this.
  11. Overstressed

    Overstressed Senior Member

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  12. alex3619

    alex3619 Senior Member

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    Determing a healthy sequence may be very problematic. The question itself is problematic. What is healthy?

    @Waverunner is right with respect to CFS or other specific diseases, first determine the bad genes, then determine the genes of people who do not get the disease.

    However the classic counter-example is sickle cell anemia. You can determine that those without the sickle cell gene wont get the disease, and are healthier. So you could go into a population and eradicate the gene. However this gene confers near immunity to malaria. Now you will change people so they are vulnerable to malaria. Malaria is also very painful, and also kills.

    Actually its more complicated than that. Those who are only sickle cell carriers (a single copy of the gene) wont get sickle cell anemia, but are resistant to malaria. Those with two copies are near immune to malaria, but suffer severely from sickle cell anemia. This gene protects the population, but at a heavy price.

    Similar issues occur with detox polymorphisms in the liver. Some are not so good with some chemicals, so you get sick. However in many cases being bad at one chemical makes them better at another. If you remove one variant from the population, then you might put everyone at risk.

    Its easier if someone has a severe disease though, like full sickle cell anemia or ME. The problems destroy their lives, and might kill them. So I think most who are afflicted would be happy to take a risk with another gene version.

    I think there are probably many different genes involved leading to ME. Some of those will be nothing but harmful, but some will have benefits too. This is an area that will need very much research once the technology is mature enough to fix adults.

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