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Gene Therapy Cures "Bubble Boy"
bofh31337 writes "NewScientist is reporting that Welsh boy Rhys Evans has been cured of the fatal severe combined immunodeficiency ("bubble boy") disease. The medical team, lead by Adrian Thrasher, was able to take the stem cells that give rise to immune cells from his bone marrow and add a normal copy of the gene to the stem cell using a retro virus. Seven months after treatment, Rhys was cured."
Sadly there is no cure for the people who got sick after seeing the movie "Bubble Boy"
http://www.kubuntu.org/
...science can answer the Moors/Moops riddle which has plauged mankind since the dark ages!
The potential power of stem cell research is clearly evident in this case. My question, not to troll, is whether this type of research could have been possible/allowed in the US. AFAIK, the laws in the states allow a restrictive amount of stem cell research -- would this have been enough for similar treatment here?
The Guardian, and Yahoo
My other sig is also a
The biggest problem with gene therapy is that long term expression of the target gene has been difficult to achieve. The inserted sequence, depending on the gene carrier, may or may not be inserted in to host genome. Actual insertion into the host genome is undesirable because of possible malignant transformation (insertion of the target sequence disrupts the function of a tumor supressor protein, or turns on a pro-tumor protein, etc.). Existing as a genetic sequence outside of the genome proper has also failed to achieve more than temporary expression of the desired protein.
This article describes a technique to increase the effiency of the transfer of a therapeutic gene sequence into a target cell. It does nothing to address the biggest stumbling block of gene therapy. While this is sexycool news, being cured for 3 or 7 months doesn't mean being cured for life.
Claimer: IAAMD
I don't mean to be a downer. We're just a loooong way off from real gene therapy.
Fermat's other theorem: "I have a simple proof, but I can't write it down as I fear it's a DMCA violation to discuss it"
Why is it that no matter what things humans do to the earth (good or bad), the ONLY time we're "playing God" is when we fiddle with genes. Very arbitrary criterion if you ask me. Did not God create the trees and the animals? Why when we destroy or create these things then are we not "playing God". It seems a bit illogical to me.
Retroviruses are being investigated for 3 reasons:
1) They can be used as vectors to transport genetic information into a host cell.
2) Reverse transcriptase can be used to isolate DNA sequences from a mRNA chain so that the gene can be manipulated through bioengineering techniques.
3) To find a way to genetically engineer a cure for AIDS. If the action of reverse transcriptase can be halted somehow, the HIV virus will have no way to spread its harm through the body and millions of lives could be saved.
more info
While this was going on there is a couple in California that is hopeing (as in activly looked for sperm donner who was deaf) to have a kid that is deaf so that he will be like the rest of the family (minus the cats). So while we have gene experaments going on to inhance the lives of people and potentialy bring a brave new world kind of classism effect [BadThing(TM)] we have also got people who are actively trying to set the pace of progress back.
Ascii artist &
Remember that one of the arguments against vaccination when it was discovered, was that we shouldn't be "playing god". Eg people should just accept death by lethal contagious viruses like smallpox -- vaccination is "playing god".
Just about every significant medical discovery has been opposed with the "playing god" argument.
When vaccination was discovered, the religious community spoke out against vaccination on the same grounds -- that dying of smallpox was "god's will" and that vaccination was "playing god".
The claim is as stupid today as it was then.
X chromosomes are distinctly different from the autosomal chromosomes. No human being can live with a missing autosomal chromosome (e.g. only one chromosome 21 instead of two) -- embryos with this type of defect are miscarried so early that they are not even detected, even though embryos with three copies of an autosomal chromosome (a defect arising from the same mistake in meiosis which causes the loss of an autosomal chromosome in some embryos) are detected -- some even live to adulthood (Down syndrome). On the other hand, all human beings can be said (in general) to have only one X chromosome; in females, one X chromosome is almost completely inactivated in each cell.
Well technically, manipulating all reality through the power of will alone is a pretty good functional definition of what it means to BE God. Only important thing missing is knowing everything, but that probably comes along with all events playing out according to one's will.
As you no doubt know, "playing god" is what the disdainful call it every time man gains control over an aspect of reality that was previously ruled by chance/God alone.
Since God created us with intellect, reason and the ability to learn moral judgment, I for one, believe that we should "play God". Man is created in the image of God, and if we are to fulfill that destiny than it means learning to act with as much wisdom, knowledge and moral judgment as humanly possible, which certainly includes scientific exploration.
Name me one thing in nature we fully understand. Name me one thing.
We don't know, *for sure* how atoms work or are built. We don't know if there is a 5th repulsive force in nature. There's lots we don't know..
But what we do know.... To our knowledge, this therapy may help a guy who's *never* had a chance to go out into real life. Maybe it'll give him cancer in 30 years. Maybe it won't.. But just because it might possibly be catastrophic doesn't mean that nothing should be done.
That way leads to stagnation and helplessness. We don't know and can't know. That is why this so-called 'precautionary principal', that something must be proved 'safe' before it can be used or sold is garbage. We can't know and won't know for *sure* anything.
I'm not suggesting for a minute that we stop. I am truly in awe of what they have accomplished, and the incredible potential for improving human lives. I thought this was an exciting story, and I am happy for the boys who suffer from this disease. Maybe it's because I'm more of a physical sciences kind of guy, but thought of being able to mainipulate individual human genes, effectively retroactively as I understood this, is just mind-boggling. If we're advanced enough to pull this off, are there any limits to what we can do?
And that is where the negative side of my comment comes from. What are the limits to what we can do, and (rhetorically) are we up to the responsibilty? The answer is "no" - though the prospects for good are unlimited, some will abuse this technology. It's the inevitable cloud that accompanies the silver lining.
In my opinion, that's part of the price we pay for advancing. Genetic manipulation seems much like our first steps into atomic power (another subject that provoked fears of "playing God"). It is far more revolutionary than medications or cutting trees or most of the other ways we manipulate our world. These other things can have tremendous cumulative effect due to scale, but their potential individually is fairly narrow and limited. A new drug may heal - or hurt - a few individuals, but it can't change the shape of the human race.
Genetic manipulation is different. It can literally change the face of humanity. The potential for good is awesome, but it will come with a price. And that's the risk we accept every time we move forward.
Again, sorry for provoking a religious discussion. My use of "playing God" was only meant as a metaphor for the power and potential of this development.
God gives us everything. At least that's what I belive. If god does not want us to know something, he won't let us. That's my opinion. So long as any gene research does not result in a death or crippling some one or for the sake of better boobs, I think it's ok in my opinion. I personally would have loved some of this stuff to be found out years ago because now I may have a grandma to talk to and her talk back instead of visiting someone who can just listen to me. I am not sure what she had, but what I do know is it messed her brain up and she's almost not like grandma. All of her mental functions are ok, it's just that she doesn't have much control of her muscles. It started as a flayling of hands....for a time they thought it might have been Parkinsons, but it wasn't. Now you have to be careful how close you get to her sometimes cuz she could accidently slap ya. Not funny and she doesn't mean to do it, but if they had figured out how to fix this in time, she'd be mostly normal (even if she's old). I want to WALK with my grandma and have fun with her like I did when I was a kid/teenager. My son will only know his great grandma as some old lady in a bed. If any religious zealot wants to keep this from people who truely need it, then to heck with em! How many years more of Michael J Fox's acting would we have if they could reverse his parkinsons? I think it's right as long as we are fixing something. Doing genetic alterations in the womb is something that should not be done and also gene alterations just for better looking kids is a bad thing too. Genetic alteration to fix something that could hurt someone all of their life, well, I feel a bit different about that cuz if this stuff would have happened sooner, I would probably still have a vibrant grandma instead of a invalid grandma.
Gorkman
Nope -- see the Straight Dope column answering that question.
/. If the government wants us to respect the law, it should set a better example.