gene therapy not possible for this alien

L M Ashton

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I have an alien with a genetic disorder that I need to be either untreatable, extremely risky, or extremely expensive.

This alien has been cast out from her xenophobic species, so she'll get no help from them. However, the aliens she works with are highly advanced with science & tech with a great deal of resources at their disposal. They're far advanced of present-day human tech and they're a space-faring scientifically-minded group of aliens from a fair number of planets/solar systems.

My uneducated-in-this-area mind would think that gene therapy should be fairly standard.

What are some possible plausible reasons for gene therapy for her to be impossible, extremely risky, or extremely expensive?
 

Treehouseman

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It's too close a gene location to a "stop codon" and runs the risk of deleting it during the gene transfer? Causing runaway alien cancer?

Or it's in another artificial organelle they've lost the tech patent to (like a mitochondria) and there is no suitable viral vector to implant the required genes?
 

frimble3

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She's the only one of her species that they've ever had much to do with, so they'd have to use her substandard DNA as a template, or, even if they're advanced, it's the old 'orphan disease' problem: too expensive and too much effort to do all the work to make a custom cure for one customer. If you want her to live, have one scientist be persuaded to take her on as a special project, if not, well, nice knowing her.
 

L M Ashton

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Treehouseman, thanks for the ideas. I now have research...

Frimble, would that be the case even amongst aliens with advanced technology? Yes, she's the only one of her kind they have genetic material for, but is that enough?
 

thepicpic

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Could she simply not be receptive to it? That's how I'm working around the gene therapy in my 'verse.
 

Debbie V

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Gene therapy is great at treating single-gene cancers, but some diseases involve more than one gene mutation. Maybe the disease itself is too complex for gene therapy alone.

Maybe the species doing the work doesn't have enough understanding or quite the right tools for treating the species with the disease. This will depend on how different the species are. Developing those two things can take time, perhaps even more time than the patient has.
 

flapperphilosopher

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Maybe there are different mutations of the genetic disorder? This is the case with cystic fibrosis, which is genetic--there's a whole bunch of different genes that can have the mutation. They're in the process of developing gene therapies to treat it, but they have to target them towards one gene at a time. The one they've come out with so far treats a mutation affecting something like 5% of CF-ers, which is swell for people with that mutation, but does nothing for the people with the CF mutation on other genes. So of course now they're working on the most common one, which of course builds from what they have but still involves years more of work, then presumably they'll go on to the next one, etc. The rarest mutations are probably going to be last, if they're done at all. This gene therapy still isn't any kind of cure, because it's a complex illness--I'm not sure of all the details but if you are keen on looking into it the drug is called Kalydeco, someone might explain somewhere why it's limited. But even if your aliens can cure genetic diseases through gene therapy, there's still going to be the issue of different genes needing to be targeted separately. If they've never addressed that one--either because it's rare overall, rare in their population, or non-existant in their population--they probably can't just whip a treatment up, no matter how high-tech they are. They'd have to do it specially, and they'd have to have a good reason to. One random alien from another species probably wouldn't be worth the time and cost, unless she's super important or they're super altruistic.
 
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TellMeAStory

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My husband, recently retired from the field of gene therapy suggests a disease in which many genes have been mutated. You could set up a scenario in which one or more of those genes is/are essential for other functions.

Another way to do it would be to have the mutations complementary in a way that actually allowed the cell to function. Correction of one of those genes would damage that complementarity, resulting in death.

There's a precedence for that: repairing the mutated gene that causes sickle cell increases vulnerability to malaria.
 

L M Ashton

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Thank you all for your comments. I think I have enough to go on for now. :)

flapperphilosopher, yeah, I'm aware of that sort of thing. I don't think I knew that CF was a variety of genetic disorders, but I have one of my own - Ehlers Danlos Syndrome, which is a genetic collagen defect. There are six major types and five minors (or something like that). The genes responsible for some have been identified, but not all. My type is one that hasn't been identified even though it's the most common. And there's no treatment for any of its types except for the individual symptoms/diseases caused by the EDS. And then there's one of my brothers, who has a genetic defect of a different sort that resulted in blindness, but his particular flavour is so rare that there *might* be ten people on the entire planet with it, so his defect is, well, never going to be fixed. Orphan diseases suck.

I'll look up kalydeco. More information is better. :D

TellMeAStory, you've got a great idea, and it fits in very well with what I need. Thank you so much. :) I like the idea of many genetic defects combined with how they interact with each other. And throw in some epigenetics for good measure, and I think I've got this covered. :)
 

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Given the extraordinary ability of carbon and hydrogen to form a vast variety of chemical bonds and molecules, there's no reason to believe alien life forms would even have "genes" or DNA. They might well be based on some entirely different chemical construction. In particular, right-handed amino acids. Amino acids are the building blocks of protein molecules, and subsequently of Earthly biotic DNA, but all DNA known, from any life form on Earth, is, as I understand it, based on left-handed amino acids. Right-handed amino acids, the mirror images of the lefties, are known to exist, and can be constructed via inorganic processes in space. That we Earth organisms are all lefties is strong evidence of a single origin for life on this planet. No reason somewhere else wouldn't work differently.

caw
 

frimble3

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Frimble, would that be the case even amongst aliens with advanced technology? Yes, she's the only one of her kind they have genetic material for, but is that enough?
What part? The genetic material thing? I think the other posters, discussing the way that many genes might play a part in the disease, kind of explain it: how are these advanced aliens going to know what the 'correct' DNA is supposed to look like?
 

blacbird

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I think I ought to add here that the concept of living organisms being based on something other than carbon-hydrogen chemistry is vanishingly remote. H is by far the most abundant element in the universe, and C is extremely abundant as well. No element has carbon's flexibility of chemical bonding potential combined with the potential for energetic reactions. C-H chemistry is why we're all here debating fantasy ideas about alien life forms.

caw