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Gene Therapy Corrects Hemophilia in Mice and Dogs
FiReaNGeL writes "Researchers successfully corrected hemophilia, the oldest known hereditary bleeding disorder, in dogs and mice. They introduced the gene coding for clotting factor VIII, which is defective in type A hemophilia, in the liver of newborn animals. All animals achieved expression of the clotting factor, averaging 139 and 115 percent of normal factor VIII activity and are stable after more than a year. The treatment targeted newborns exclusively for two reasons; their liver is still developing and their immune system is immature, facilitating the gene transfer. Researchers plan to perform tests in higher primates in the next few years. A summary of the research and the reference to the original paper are available."
I'll probably bleed to death before they release a real cure.
BeneFix Recombinant Factor products cost about $1000 per 10CC, with a typical dose being 40CC. Typically a patient receives three treatments a day for two weeks after surgery or an accident. That's $168,000. There is just too much profit in these drugs for hemophilia to ever be cured.
Of course, that cash amount doesn't look nice to potential employers. I don't participate in anything now since I can't afford this stuff.
I've considered living out in the woods, but I'd probably injure myself and die a slow and horrible death.
In conclusion, I'd like to thank Benefix for saving my life and curse them for making my ass unemployable with thier overpriced medicine.
that is because there is another bleeding disorder called von Willibrands disease. The twoa re similar but not the same. One is the failure of the blood to clot which I think is von Willibrands disease, and the other is taking long time to clot which is haemophilia. Those descriptions could be wrong, but there is certainly more than one blood clotting disorder.
"I just can't sit while people are saying nonsense in a meeting without saying it's nonsense" J Watson, Sci Am 288:(4)51
As for the drug companies, there will always be another disease/condition to develop drugs for and they could invest in gene-therapy developers.
The imminent collapse of space and time is just the Universe's way of hugging you.
Perhaps dog breeders might do this, but mice?
Yes, mice. If you ever want to retire early and comfortable, breed a stable, genetically-deficient blood-line of mice. You'll make a killing.
Getting mice with any kind of genetic disorder would not be too difficult, since mice are grown for laboratory purposes all over and if a suitable bacch is not found then the scientists could relatively easily introduce the genetic defect to the mice. It's been done before.
Dogs are a bit trickier, but as lab animals they are probably not that different from the mice.
Scientists don't just go to pet shops to buy the lab animals. They are usually bred especially for the purpose of lab experiments.
The drug industry is fighting food "supplements" because drugs are required to prove that they are safe and effective before they can be sold, and food supplements are not. You may be able to get actual help from a food supplement, or you might not. Unlike an FDA-approved drug, neither the supplement nor its mode of administration have been tested for safety and efficacy in double-blind protocols. People may make claims for the wonderful effects of different supplements, but there is literally nothing to distinguish their experiences from the body fixing itself or the placebo effect.
There have been examples of calcium supplements which contained lead. There are all kinds of supplements which contain widely varying amounts of the supposed active ingredients, including none at all. Quality control is very easy for supplement makers. If you have no standards for your product, everything you produce will meet them. The buyer may be getting nothing, or may be taking an overdose - unless the actual stuff they're taking is assayed, nobody is going to know.
I personally am taking vitamin and mineral supplements, but they have stated (and presumably assayed) amounts of the various substances therein. I can total the amounts I'm taking and verify that I'm neither deficient nor in the overdose range (both of which are bad). I started taking vitamin D after I found a couple articles in Science News pointing to research showing that many people are D-deficient and that this causes loss of muscle strength as well as bone density - and found that my back pain cured itself after about a week of taking 600 IU per day (which was a totally unexpected result but which made me very happy nevertheless). I'm currently up to 800 IU just to make sure; this is still well under the toxic dose. I don't get a heck of a lot of sun for most of the year and I can't drink milk, so I don't get much otherwise.
If we wanted to make sure that supplements were actually doing anything (let alone what they are claimed to do), we would require:
Without spending the money ($millions) to do this for every supplement on the market, there is absolutely no proof that they do anything
Should the government be funding such trials? HELL, YES! And we should be using the best of modern gene science to find out why everything works, so we can determine who needs what interventions. If you think about all the money that could be saved if we could hold off or prevent problems using vitamins and herbs instead of treating them with drugs, the nation would be far ahead. But without those trials to give us real reliable knowledge, everything might as well be snake-oil sold by hucksters.
http://www.answers.com/menstruation&r=67
menstruation (mn'str-'shn) pronunciation
n.
The process or an instance of discharging the menses.
http://www.answers.com/menses&r=67
menses (mn'sz) pronunciation
pl.n. (used with a sing. or pl. verb)
The monthly flow of blood and cellular debris from the uterus that begins at puberty in women and the females of other primates. In women, menses ceases at menopause. Also called catamenia.
[Latin mnss, pl. of mnsis, month.]
Yes I know you were trying to be funny, but that question was just begging to be answered.