In a 1st, Doctors In US Use CRISPR Tool To Treat Patient With Genetic Disorder (npr.org) 67
An anonymous reader quotes a report from NPR: For the first time, doctors in the U.S. have used the powerful gene-editing technique CRISPR to try to treat a patient with a genetic disorder. "It is just amazing how far things have come," says Victoria Gray, 34, of Forest, Miss. "It is wonderful," she told NPR in an exclusive interview after undergoing the landmark treatment for sickle cell disease. Gray is the first patient ever to be publicly identified as being involved in a study testing the use of CRISPR for a genetic disease. "I always had hoped that something will come along," she says from a hospital bed at the Sarah Cannon Research Institute in Nashville, Tenn., where she received an infusion of billions of genetically modified cells. "It's a good time to get healed." But it probably will take months, if not years, of careful monitoring of Gray and other patients before doctors know whether the treatment is safe and how well it might be helping patients. "For the study, doctors are using cells taken from patients' own bone marrow that have been genetically modified with CRISPR to make them produce a protein that is usually only made by fetuses and by babies for a short time following birth," the report adds. "The hope is this protein will compensate for the defective protein that causes sickle cell disease and will enable patients to live normally for the rest of their lives."
Frosty Piss (Score:1)
Ideal disorder to treat (Score:3)
This seems like an ideal disorder to treat. The progress of the disease is relatively slow. There are other treatments. The genes involved are well identified, and natural to human beings. There is strong emotional appeal to successfully treating lethal cancers. And it's a relatively common form of cancer, so the pool of patients who might benefit is large.
For medical and epidemiological safety, however, I do think we need to be very cautious. Careless doctors, and exhausted interns or overworked technicians, will make mistakes and take short cuts. I'm concerned that casual editing genes for fertility or to treat genetic diseases for our descendants could have devastating consequences on the patients or resulting children. Genetic censorship is already in place for the disorder of being born female, or having Down's syndrome: it was one of the strongest reasons for the growth amniocentesis in China for the last generation.
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Funny thing about the Down Syndrome test: A friend of mine was pregnant last year and got the Down Syndrome test due to some risk factors. Now this test was supposed to be absolute positive or absolute negative. What they gave her was a 60% risk. (Might be because she is a lawyer. That apparently comes with all kind of entertaining extra information when you are a patient...)
This rather strongly indicates that test results given to parents before were flawed and that does not speak well of this industry. Af
Re:Ideal disorder to treat (Score:4, Insightful)
That is unusual. A result like that usually just means 'test again with amniocentesis so we can be sure.'
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You think something that millions of people do in every election is unusual?
Re:Ideal disorder to treat (Score:4, Insightful)
A friend of mine was pregnant last year and got the Down Syndrome test due to some risk factors. Now this test was supposed to be absolute positive or absolute negative. What they gave her was a 60% risk. [...] This rather strongly indicates that test results given to parents before were flawed
The test results were most likely not flawed. Either the gynecologist was not properly trained or your friend didn't understand or remember what they told her. Noninvasive prenatal screening has always given a percentage risk of having down syndrome. Your results will either be "positive" (>= 98.6% chance of the chromosomal disorder), "negative" (>= 98.6% of not having it), or somewhere in between. Your friend was likely just in the somewhere in between category.
Families who want a diagnosis and do not receive a positive or negative result can resort to a more invasive procedure such as chorionic villus sampling or amniocentesis. These procedures have a low risk of causing miscarriage (less than 1% chance) but are 99% accurate in diagnosing Down Syndrome.
Even if your friend had one of these procedures and they couldn't determine accurately, it still doesn't show the test is flawed. People see 99% accurate and sometimes treat it as if it is 100%. With nearly 4 million babies born per year there is plenty of room for anecdotes about false positives and false negatives even with a 99% accuracy.
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I'm no oncologist, but I'm pretty sure sickle cell disease is not a cancer.
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Oh, Dear. You're quite right. I'm quite embarrassed.
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I was going to say this seems like an odd choice for something to start with. Sickle cell disease isn't cancer. The red blood cells are malformed and die early, so you end up chronically anemic. There are effective treatments, and most patients live full lives.
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This seems like an ideal disorder to treat. The progress of the disease is relatively slow. There are other treatments. The genes involved are well identified, and natural to human beings.
And best of all, sickle cell is an African-American disease. Let's see the No GMO liberals try to protest this application of CRISPR.
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It's actually more of a tropical disease, which includes much of Africa. Scientists believe that it's an adaptation to resist malaria. If you're only concerned with the U.S. then, yes, it's most prevalent among African Americans. However, that's not the same as "an African-American disease."
Most anti-GMO people are concerned with unintended consequences. They also tend to overlap with the "organic" types, and they dislike that the most common genetic modification is to make herbicide resistant plants and th
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It's all fun and games until it becomes the next asbestos or leaded gasoline. History teaches us that as a collective we tend to underestimate the possible outcomes until it's too late when it benefits us.
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Emotionally-driven people will start shouting "Tuskegee" or something similar. Don't force your shitlord reason and evidence on them!
The real issue is "consenting adult", not collectivist tags. I, for one, am deeply grateful for this woman's bravery and willingness to be experimented on. You can be damn sure the IRB required full and metaphysically complete consent.
Re:Cure for liberalism? (Score:4)
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I wonder if this technique could be used to change the expression of genes related to personality, and if so, could it eventually be used to actually change someone's personality?
Yes. And no. Anybody who has raised puppies can tell you that genes affect personality quite heavily. But once you have an adult, the gene expression that built that brain isn't going to change that brain very much if the genes are altered. All the growth is already finished. For the longest time it was thought that adult mammals never grew new brain cells. We now know that's nonsense, but we do know the rate of (normal) growth is extremely low, which leaves very little room for a gene therapy to chan
Might also have "wonderful" side effects (Score:3)
After all, understanding the information encoded in the genes is in its infancy. Messing with one part to have one effect may well cause severe negative effects someplace else and that may well be decades and/or generations removed. That is not to say, do not try it when there are severe problems that can be fixed. I am all for that. But be very careful, and by all means make sure these patients do not have children (who would most decidedly have not given informed consent here) until things are understood much better.
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They aren't changing all of their DNA. Just some the DNA of some bone marrow cells.
So there's no chance of this being passed on to the next generation.
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That is what the theory says. Medical theory has proven to be dicey and frequently wrong in the past. Hence care is advised.
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They do improve the chances by removing the cells to be altered from the body first.
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There is a slight difference to have a known risk on one side and some risk that is not quantifiable on the other. Anybody that knows they may inherit some genetic defect should carefully think about whether they can justify that. But they will have information about what they might inflict on another human being, i.e. there will be a basis for that decision. With genetic manipulation, they will just not know, there is too little data.
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In this case, they minimize the risk by only altering the patients marrow. If the same gene has any effect in other tissue, it won't matter because the other tissue is unaltered. The most concerning would be alterations that could be passed to children, of course, that too isn't an issue here since the germ line is unaltered.
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That is what they think things work like. And there is a reasonable chance they are correct.
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"I'm, I'm simply saying that life, uh... finds a way."
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Modifying somatic cells wouldn't change the sex cells so it wouldn't have any effect on potential children. This actually might be a downside as it wouldn't allow us to eliminate genetic diseases in this way, we would just have to treat every generation that inherits the disease.
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Do you understand the difference between modifying cells in a consenting adult vs. creating a genetically modified baby?
Hydroxyurea (Score:3)
There is already a fair amount of data from administering hydroxyurea to induce production of fetal hemoglobin [bloodjournal.org], the same as the goal in TFA.
Species Dysphoria (Score:2)
I will be happy when (Score:2)
they make most of the idiots on /. either braindead or smart.