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Gene Therapy Corrects Hemophilia in Mice and Dogs

timothy posted more than 9 years ago | from the simple-chronic-halitosis dept.

Biotech 42

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."

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Anonymous Coward | more than 9 years ago | (#12276253)


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gnaa fp (-1)

Anonymous Coward | more than 9 years ago | (#12276254)

gnaa at the top of the page
penisbird at the head of the game
you fail it penisbird

woo (-1)

Anonymous Coward | more than 9 years ago | (#12276271)

"and remember kids, gene therapy + firmware = genetic memories" -buddha

Re:woo (-1, Offtopic)

Anonymous Coward | more than 9 years ago | (#12276688)

damn it someone put that as their sig!

I thought it said "homophilia" (-1, Offtopic)

Anonymous Coward | more than 9 years ago | (#12276281)

and then I thought, how long until someone GNAA trolls this?

fixing hemophilia is nice.. (-1, Offtopic)

Anonymous Coward | more than 9 years ago | (#12276283)

..but soon they'll have a fix for pedophilia!

WHats sad is.... (5, Insightful)

Creepy Crawler (680178) | more than 9 years ago | (#12276313)

Most drug companies will petition AGAINST these types of treatments for the sake of "medical scrutiny not up to standard".

Go take a look at [] and take a look how Type 1 IDDM can be cured by using YOUR OWN BODY PARTS.

Now, why would drug companies NOT want to do away with insulin dependancies? Well, soo much for selling:

Testing Kits
Monitoring Equipment
and much more.....

Thats one hell of a profit margin that would be lost if even 1 person was cured of insulin dependancy. Now think of if all were...

Re:WHats sad is.... (0)

Anonymous Coward | more than 9 years ago | (#12276370)

yeh it sucks that the fda is in the pockets of the major drug companies

Re:WHats sad is.... (-1, Troll)

Creepy Crawler (680178) | more than 9 years ago | (#12276463)

It is the FDA, but not just the FDA.

Right now, Diabetes is on the track of being "cured" by use of embryonic stem cell research. Be aware, that a "Cure" like this hooks you to cyclosporine for the rest of your life, lest you eject and destroy those embryonic 'non-self' cells.

Its yet another way for the pro-choicers to get a foot in the door. Let alone I dont consider anything that hooks me to a drug for the rest of my life a 'cure' of anything.

Re:WHats sad is.... (1)

phloydphreak (691922) | more than 9 years ago | (#12276455)

I doubt CORPORATE AMERICA(tm) would directly oppose such a development, if only because the bad PR which would arise from said opposition. The continuation of scientific development is the lifeblood of the American Market; especially the upgrades required to stay competative.

Re:WHats sad is.... (4, Insightful)

Creepy Crawler (680178) | more than 9 years ago | (#12276493)

Then you fail to grasp corporate language.

When a group of well-paid 'scientists' appears in front of a FDA panel for phase 3 testing saying:

"We feel, after going through our and their data, that this proposed treatment has unforseen detrimental effects and must be delayed for further study"

And of course, unlimited delays mean no treatment. And more profit for the insulin profit-derived makers.

Haemophilia (3, Funny)

rookworm (822550) | more than 9 years ago | (#12276358)

the oldest known hereditary bleeding disorder

Aww nuts, I thought it was that other hereditary bleeding disorder.

Re:Haemophilia (4, Informative)

Zugok (17194) | more than 9 years ago | (#12277049)

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.

Re:Haemophilia (-1, Troll)

Anonymous Coward | more than 9 years ago | (#12278361)

Menstruation... He was talking about menstruation.

Re:Haemophilia (2, Funny)

rookworm (822550) | more than 9 years ago | (#12278484)

That is merely a symptom, not the disease. (ducks)

I have a question ... (1)

some guy I know (229718) | more than 9 years ago | (#12278505)

Why is it called menstruation if only women get it?

Re:I have a question ... (3, Informative)

ikkonoishi (674762) | more than 9 years ago | (#12298976) []
menstruation (mn'str-'shn) pronunciation

The process or an instance of discharging the menses. []
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.

Re:Haemophilia (1)

PygmyShrew (618310) | more than 9 years ago | (#12283056)

You'd know this kind of thing if you subscribed to Bleeder's Digest.

Speaking as a hemophiliac (type B)... (5, Informative)

Xenkar (580240) | more than 9 years ago | (#12276519)

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.

Re:Speaking as a hemophiliac (type B)... (1)

rookworm (822550) | more than 9 years ago | (#12276698)

I thought such treatments required huge quantities of donor blood, hence such a large price. Is this correct?

Re:Speaking as a hemophiliac (type B)... (5, Interesting)

Xenkar (580240) | more than 9 years ago | (#12276887)

Actually the Benefix Recombinant Factor products are made from genetically altered hamsters. The process of harvesting the factor kills the rodents so my existence is the bane of any animal rights advocates.

This also means that I won't get HIV or any other nasty blood disease. Most hemophiliacs don't feel safe using donor blood due to the HIV outbreaks of the Early 80's. About 44% of hemophiliacs at the time were infected.

The blood supply is supposedly perfectly safe now, but I wouldn't touch that stuff even if it'd save my life.

My uneasiness comes from the thought that someone could get infected with HIV and go donate blood before any detectable amount of HIV anti-bodies form in the blood. They actually test for the presence of the anti-bodies instead of the actual virus.

Re:Speaking as a hemophiliac (type B)... (1)

daveewart (66895) | more than 9 years ago | (#12280445)

They actually test for the presence of the anti-bodies instead of the actual virus.

That's because the antibody test is simpler, quicker and cheaper and usually very accurate. Not saying it's Right, just an observation.

Re:Speaking as a hemophiliac (type B)... (1)

LoneGunner (636894) | more than 9 years ago | (#12281131)

A cousin of mine who was a hemophiliac contracted HIV from donor blood. They discovered he had it after tests run by the company making the treatments. They had discovered a bad batch had been released for use. He was around 18-20 at the time and lived to about 25-26 before his immune system succumbed to the HIV. That was about 15 years ago.

Re:Speaking as a hemophiliac (type B)... (1)

Scott7477 (785439) | more than 9 years ago | (#12278576)

Just out of curiosity, do you know what it costs the manufacturer to actually produce this stuff? I am imagining maybe $100 per cc at most. As you say, the pricing is obscene. The manufacturer probably would cover their research costs after 5 years at the most.

Re:Speaking as a hemophiliac (type B)... (1)

Martin Blank (154261) | more than 9 years ago | (#12292362)

Just wondering... Do you have any information to back up this guess? I'm looking around, and I can't find anything. I know BeneFIX is manufactured by Wyeth, and that sales have been relatively good for the last few years, but that's about all I can find on it.

Re:Speaking as a hemophiliac (type B)... (1)

Scott7477 (785439) | more than 8 years ago | (#12312601)

I found this link: tml [] . It says that the price in NYC is $.76 per unit. It looks like my guess may have been off base. Apparently this particular medication is somewhat difficult to produce.

Re:Speaking as a hemophiliac (type B)... (2, Informative)

bobster45 (816998) | more than 9 years ago | (#12286182)

I lost a very wonderful friend, (that had hemophilia) due to the AIDS complications from infected clotting factor. He had to introveniously inject these clotting factor all of his life. He was living in America and was transfered to Scotland on a job assignment. It turns out his brother had hemophilia also and had been diagnosed as having an unusual brain tumor that was linked to AIDS. His brother was remembered to have told the doctor when he was told of having AIDS that he had never had intercourse with anyone.Obviously his AIDS had been linked to the clotting factor he had to inject. Anyway my friend came back to America (to be by his brother's side) from Scotland where all the blood products were screened for HIV. From the Americaan blood products he too soon became infected and after bouts of totally weird infections succomed to pneumonia.

I was so upset to learn that the tainted blood products he had to inject were the cause of his AIDS and low T-cell count. The cure in this case killed him rather than the diesease!

To hear that this new developement is on track is very wonderful news indeed, yet a bit late for many. I hope this proves to be a viable alternative and potential cure for those with the affliction of this debilitating diesease.

I pray that the pharmaceutical companys are benevolent enough to make and develop these products to at least lessen the chance of being infected with horrendous complications that are associated with human blood products.

I didn't even know... (-1, Offtopic)

Drunken_Jackass (325938) | more than 9 years ago | (#12276897)

...that there were gay mice and dogs.

That's weird.

One problem... (1, Insightful)

Game Genie (656324) | more than 9 years ago | (#12277723)

Where did they find enough hemophilic mice and dogs? I wouldn't think there is much screening for such genetic disorders in animals. Perhaps dog breeders might do this, but mice? Or do they just breed them on purpose from hemophiliac parents?

Re:One problem... (1, Informative)

Anonymous Coward | more than 9 years ago | (#12279339)

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.

Re:One problem... (4, Informative)

valkoinen (81260) | more than 9 years ago | (#12279636)

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.

Not usually an optimist... (2, Informative)

Mistress.Erin (875686) | more than 9 years ago | (#12278912)

I'm not usually an optimist, but how can you just throw away the hope of a real cure because of lost profits? If this were true, we'd still be using leech courses. Think of how much money those leech farmers lost when penicillin was discovered! This condition has been greatly researched and now we're seeing results. It was even discussed in a biochemistry 100 level class here at my university. My first response to this article was to be thankful.

As for the drug companies, there will always be another disease/condition to develop drugs for and they could invest in gene-therapy developers.

Re:Not usually an optimist... (2, Insightful)

cowgoesmoo2004 (762366) | more than 9 years ago | (#12280887)

I think you are being a bit naive.

Food supplements are making inroads into various health issues. However, the drug industry is fighting them tooth and nail with the help of the FDA -- because they hurt profits.

I'm not a conspiracy theorist wacko, just look at the hatchet job they attempted on the Life Extension Foundation [] . I don't have the exact link, you'll have to do some digging to find it.

The real issue is money for testing (5, Informative)

Anonymous Coward | more than 9 years ago | (#12283428)

(Posting AC 'cause I don't want to cancel my good mods above...)

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:
  1. Supplements of known and consistent composition
  2. Used in double-blind trials

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.

Re:It's Ceding of Authority (1)

cowgoesmoo2004 (762366) | more than 9 years ago | (#12284575)

You are partially correct. However, it is a real stretch to say their is no proof or that there is no research.

Have you ever done a search on PubMed? There are all kinds of studies being done -- random double blind and so on with strong statistical results to draw from in many cases.

I will agree that people often fall prey to hype, but is it really the job of the government to act as our nanny? What if you are sick and traditional medicine does not have much to offer? Should the government deny you the opportunity to try non-traditional means?

Health is just an area that people treat as a sacred cow. It is not right that in all other areas we are allowed to make our own decisions, supposedly, but that in health we are forced to let others decide what is better for us.

I would propose we disallow baseless claims and hype, which is really just truth in advertising, instead of disallowing the sale of products to those that want them.

One more thing (0)

Anonymous Coward | more than 9 years ago | (#12283601)

One reason that supplement-makers don't perform such tests is that one cannot patent herbs and the like, and without a way to get a commensurate revenue stream from the product there is no money to perform the kind of double-blind testing that is done with drugs. (If anyone can sell e.g. echinacea, you can't raise your price to recover the cost of testing from your sales.)

Re:Not usually an optimist... (0)

Anonymous Coward | more than 9 years ago | (#12285625)

Well, I wouldn't really view "Life Extension Foundation" as having much credibility. Even their name is playing on people's desires for longer life and fears of death. There are plenty of hacks out there. And some of these "suppliments" are actually harmful because they are packaged in too large of quantities. Despite what these hacks will tell you about "life extension" to turn a profit, these things might actually be killing you or doing nothing at all other than lightening your wallet.

See the link: itamins .htm

Re:Not usually an optimist... (1)

WillAffleckUW (858324) | more than 9 years ago | (#12283481)

I'm not usually an optimist, but how can you just throw away the hope of a real cure because of lost profits?

Ah, but that's exactly why one of the drugs we use to treat Leishmania is running out - they stopped production due to profits.

When a disease tends to impact poor third-world inhabitants, never underestimate the negative impact on drug treatment production.

Finally... (-1, Offtopic)

Anonymous Coward | more than 9 years ago | (#12278935)

A cure for Teh Gay! Oh wait, hemophilia. Ah well, there are always the good old-fashioned ways...

As a Mild Haemophiliac (3, Interesting)

khanyisa (595216) | more than 9 years ago | (#12279778)

Spelling it nicely in British English :-) Not all Haemophilia is as severe as mentioned in the article, I have 14% of the normal clotting factor and haven't had a problem for years... of course a head injury etc is still a major risk. But this is really nice research to see

As one who can spell Hæmophiliac (0)

Anonymous Coward | more than 9 years ago | (#12282953)

I think you should ph33r my mad ISO 8859-1 skillz. ;-)

(Hint: the character is æ)
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