GMO Virus: Long-term success treating Hemophilia B

[SidDithers]

One of my favorite stories is the tale of a GMO virus deployed to treat Hemophilia B:

Gene therapy for Hemophilia B

… Hemophilia B is a disease in males caused by point mutations/deletions/etc in the clotting Factor IX gene. If you dont make Factor IX, you wont clot properly, and will have all of the health issues we associate with hemophilia.

… The scientists in this paper took six Hemophilia B patients, and treated them with an Adeno-Associated Virus-8 (two at low, medium, and high doses of virus) that contained a functional copy of the Factor IX gene.

Four of them of them bumped up their Factor IX production enough to go off pharmaceutical Factor IX treatment all together (even with one of the dudes being a marathon runner!), and the two others went from needing treatments several times a week, to only needing a treatment once every couple weeks or so.

They published an update late last year:

snip

The amount of therapeutic Factor IX these patients needed (on average) dropped from 2613 IU/kg to 206. The people who got the ‘high’ dose of virus dropped that down to 92 IU/kg. They went from 15-16 ‘bleeding episodes’ a year, to one.

ONE.

And not only were these individuals lives improved, not being shackled to a hospital for IV treatments of Factor IX, not having regular ‘bleeding episodes’, their ability to live without these treatments saved $2.5 million.

http://scienceblogs.com/erv/2015/04/14/gmo-virus-long-term-success-treating-hemophilia-b/


Sid

[MineralMan]

1. We're going to see a lot more of this over time.

Using genetically modified viruses to deal with many genetic disorders is something that is being studied and tested widely, and results are very promising. This is an excellent example of how it may help people. Thanks for posting.

[FSogol]

2. K & R. n/t

[NuclearDem]

3. I await the Luddite opinion on this...

[jeff47]

4. We must label these people. They are GMOs! (nt)

[SidDithers]

6. Argle bargle Michelle Obama!!!...nt

Sid

[ProdigalJunkMail]

5. isn't this how I Am Legend starts?

except with cancer and measles???

sP

[beam me up scottie]

7. k&r

Shill.

[pnwmom]

8. That's great. All GMO's should be specifically approved by the FDA, just as that one will be

if it passes all its testing.

And anyone consuming or otherwise taking in a GMO should be able to do it after informed consent, as with all the patients in that study.

[beam me up scottie]

9. Everyone who consumes dairy products is taking in a GMO.

Cows are genetically modified organisms.

Neil deGrasse Tyson on liberal science denial and GMOs

“I don’t care if you want to label GMOs in the grocery store, but do so knowing that you will be labeling 80-90% of the food on the shelves, or go ahead and tell me you want to remove all GMOs from food, and know that the same 80-90% of food will have to be removed.”

...

“People need to know cows don’t exist in the wild, we have been modifying our food for over 10,000 years, and you need to know this stuff! You have to make informed decisions, I am not telling you to embrace GMOs, but know the facts!”

...

“The advocates keep saying ‘lets go back to nature’, but those cows don’t exist in nature, corn doesn’t exist, those red apples you love don’t exist, we genetically engineered all of that. Don’t pretend what is going on in the laboratory is fundamentally different than what is going on in agriculture.

We need to be having rational conversations about these issues, instead of just repeating your opinion.”

Tyson did say that speaking up for GMO science has made him an “Enemy of the liberals, they keep claiming I must have been paid off by Monsanto.”

http://www.patheos.com/blogs/danthropology/2014/08/neil-degrasse-tyson-on-liberal-science-denial-and-gmos/

[pnwmom]

10. Cows are not genetically engineered organisms in general,

although a specific animal could be genetically engineered.

http://agbiosafety.unl.edu/basic_genetics.shtml

What is genetic engineering?

Genetic engineering is the process of manually adding new DNA to an organism. The goal is to add one or more new traits that are not already found in that organism. Examples of genetically engineered (transgenic) organisms currently on the market include plants with resistance to some insects, plants that can tolerate herbicides, and crops with modified oil content.

[beam me up scottie]

11. MODIFIED

Who said anything about splicing?

It's GENETICALLY MODIFIED ORGANISMS, not GEO.

You don't even know what you're against, pathetic.

[pnwmom]

12. The terms are used interchangeably. n/t

[beam me up scottie]

13. Only by idiots. n/t

[pnwmom]

14. By the FDA.

http://www.fda.gov/Food/FoodScienceResearch/Biotechnology/ucm346030.htm

Genetic engineering is the name for certain methods that scientists use to introduce new traits or characteristics to an organism. For example, plants may be genetically engineered to produce characteristics to enhance the growth or nutritional profile of food crops. While these technique are sometimes referred to as "genetic modification," FDA considers “genetic engineering” to be the more precise term. Food and food ingredients from genetically engineered plants were introduced into our food supply in the 1990s.

ON EDIT:

Are you suggesting that the GMO virus that is the subject of the OP is NOT a genetically engineered organism?

[beam me up scottie]

15. Tell it to the Tyson

http://www.democraticunderground.com/10026557079

If you want to vilify GMOs and scream about labeling ALL OF THEM the label will be meaningless.

[pnwmom]

16. He's wrong. The FDA is correct.

And are you suggesting that the "GMO" virus that is the subject of the OP is NOT a genetically engineered organism? Because that is the position you have inadvertently backed yourself into.

Whether a product is a hybrid like brocollini, or the result of laboratory manipulation, I think it should be labeled. They have no problem doing so in Europe or most of the world. They can do it here.

[beam me up scottie]

17. When did you stop beating your GMO pet?

Missing the point and misrepresenting other's posts is your specialty, isn't it?

I'll wait while you show me where I made that assertion.

Then I'll address it.

[pnwmom]

18. The subject of the OP is a genetically engineered organism,

which was loosely termed a genetically modified organism. The precise term for that virus is a GEO, but I used the term GMO because that's what was used in the OP.

You then posted about genetically modified organisms while claiming that they aren't the same as genetically engineered.

I'm sorry but you're not making sense.

[beam me up scottie]

19. Fail.

And anyone consuming or otherwise taking in a GMO should be able to do it after informed consent

My responses about GMO were to address that bit of ignorance.

Up to 90% of our food is GMO.

Label all of it (that includes Elsie the cow) or none.

[pnwmom]

21. It is a false to say that 90% of our food is GMO as defined by the FDA --

containing genetically engineered ingredients. That may be true of specific foods like corn but isn't true of all of our food.

We could label GMO/GEO foods just like they do in Europe.

[beam me up scottie]

22. Who said anything about "as defined by the FDA"?

I'm with NdT on this.

"We need to be having rational conversations about these issues, instead of just repeating your opinion."

I'm not sure that's possible on DU.

The disinformation, hyperbole and scare tactics about science are right out of the Republican playbook.

[pnwmom]

23. That's more reasonable than as defined by beam me up scottie. n/t

[mmonk]

20. Bet it's not food, not in our fields and is labeled and has been through trials.

I'm tired of hearing that if you're for labeling food, you are against all GMO's and are a nut job.