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Gene Therapy Could Soon Be Approved In Europe
another random user writes
"According to the BBC, 'Europe is on the cusp of approving a gene therapy for the first time, in what would be a landmark moment for the field. ... The European Medicines Agency has recommended a therapy for a rare genetic disease which leaves people unable to properly digest fats. The European Commission will now make the final decision. The idea of gene therapy is simple: if there is a problem with part of a patient's genetic code then replace that part of the code. The reality has not been so easy. In one gene therapy trial a U.S. teenager, Jesse Gelsinger, died, and other patients have developed leukaemia. There no gene therapies available outside of a research lab in Europe or the U.S.' They have considered the use of Glybera to treat lipoprotein lipase deficiency, which leads to fat building up in the blood, abdominal pain and life-threatening pancreatitis (inflammation of the pancreas). 'The therapy uses a virus to infect muscle cells with a working copy of the gene.'"
A few years ago, Gattaca was rated the most realistic sci-fi movie by NASA. Keep that in mind everyone.
"When information is power, privacy is freedom" - Jah-Wren Ryel
Diabetes has to do with the inability to digest sugar, not fat... (sorry for my English)
Sig?
For reasons of fairness and/or perverse nationalism, I'd like to point out that the US was a bastion of eugenic progress and enthusiasm until those Germans ruined it for everyone... A few states were still sterilizing the unfit for a couple of decades after the war!
"Use a viral vector to administer the treatment" does not equate to "just tack it onto the common cold and release it into the wild." Nobody with a working genome is going to get this, because nobody's going to be injected with it unless they need to (I'm sure the cost of treatment will see to that, even without governmental restrictions). And even if they did, it wouldn't do anything - it would replace a working copy with a working copy, kind of like cut-and-pasting the same block of text into the same spot.
Everything is better with chainsaws.
You would be insane to use a fully working virus - Instead you package your pay-lode in viral proteins either within a partly functional virus, or as a replacement for a non functional one. For safety reasons the second is always preferable. The first is more risky but you would usually use a virus with no transmission ability outside of the cell line you bread it in, or in the worst case at least no ability to spread between humans. Note that neither of the TFAs differentiate at least as far as I can say so I had to look it up, and normally this info is skipped or even unintentionally lied about in the media due to ignorant journalists.
In this case "most therapeutic genes require the complete replacement of the virus's 4.8 kilobase genome"[1] as it uses a Adeno-associated virus vector[2], so likely it can not spread, this should also be the case due to the normal modifications made before use as a vector. This mean that it will act closer to a genetic drug than a actual vius, affecting the cells it enters permanently but only ever half of the offspring (does not integrate can not divide or piggyback on genomic division).
[1]http://en.wikipedia.org/wiki/Adeno-associated_virus#Advantages_and_drawbacks
[2]http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2012/07/news_detail_001574.jsp - "Glybera uses an adeno-associated virus vector as the delivery vehicle to add working copies of the LPL gene into muscle cells to enable production of the enzyme in the cells."
Gene therapy creates the opportunity to prevent Gattaca like scenarios. Within the Gattaca universe it was possible to sequence a person's DNA, but everyone was stuck with what they were born with. If you were luckily born with "good" genes, or if your parents selected for the sperm and eggs with the "best" genes with which to make a test tube baby, then your life was set. If you were born with less than "stellar" genes you were deemed inferior and discriminated against.
What is so exciting about this advance is that if you are born with a defective gene that results in illness, for a certain spectrum of genes, it is now possible to insert a non-defective version into a virus, inject that virus into muscle cells, and you are now as good as new.
This advance is about changing what genes you have at run-time, rather than being stuck with what you are born with. At the moment the changes we make are only additive, but give it time :)
So many things...
A) Infecting reproductive cells isn't quite the same as infecting muscle cells.
B) I doubt they are infecting a statistically significant number of cells in the person's body, so in the unlikely event that the virus can also infect reproductive cells it's still statistically unlikely to happen.
C) They are repairing a faulty human gene using the correct version of that human gene, as opposed to taking a gene from one species and inserting it into another
D) Only the people who elect to take part in the treatment are affected, as opposed to gene tailored crops which will find their way into your food supply no matter how hard you try
Hell, I'm not even against modded food crops (I am against the way companies like Monsanto have prosecuted people who, by some arguments, were the ones being wronged) and even I call your argument BS.
The immune system can be tricked. It is also not unstoppable. Indeed, in some cases, it is possible to seriously compromise or nearly eliminate the body's immune defenses on purpose these days. If the gene was a critical one that needed to be replaced, you'd probably put them in a bubble, compromise the immune system, administer the treatment, and then allow the immune system to regain its potency after the virus did its job. It's not risk free, but if it was the difference between possible life or certain death, it may be warranted.
And I don't think that even that will be necessary forever. Once we know enough about how things work on that level, we will probably be able to manipulate the immune system more specifically, or be able to tailor the virus to be accepted. The flip side of that is that we could then weaponize that knowledge to ensure biological weapons with almost zero immune response. Improvements in applied science can definitely be a two-edged sword.