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First UK child to receive gene therapy for fatal genetic disorder is now healthy

livescience.com

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Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#181

Post by jeffreyrogers »

bglazer wrote: post #21
SamBam wrote: post #4

The article was skimpy on the details. Can someone explain how this part works? > The new gene therapy [...] works by inserting into the body working copies of the genes that are faulty in MLD, thus restoring the ability to break down sulfatides. How does the new copy of the gene get into every existing cell that needs it? A virus?

Yes, it's a virus, specifically a lentivirus, which inserts its own genome into the cells DNA, including the therapeutic gene. Looks like they first extract bone marrow from a patient, apply the virus which adds the gene, then they put the "fixed" bone marrow cells back into the patient (probably after killing most existing bone marrow through some really unpleasant procedures). The re-inserted, treated cells then ex…

It would probably also be useful for HIV since white blood cells are produced in the bone marrow.

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#182

Post by moremetadata »

Nasrudith wrote: post #125
moremetadata wrote: post #112

Thats a new stealth weapon for the global population to watch out for now, knowing how things work in this country.

Said "stealth weapon" has been running wild for millennia. Our DNA has long been messed with by the environment, well before we knew what DNA or a virus were. The difference is now it is possible to have DNA altering viruses which aren't "almost certainly guaranteed to only do bad things to you".

Man has been trying to harness the power of nature for millennia.

King Cnut demonstrated it with the tide.

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#183

Post by arpanetus »

ChrisKnott wrote: post #171
arpanetus wrote: post #74

you can't just simply state that he's healthy without actually seeing his lifespan

You could have read the article to the third word at least, to learn that the child is a girl.

my bad, I didn't read the article, just followed the title

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#184

Post by arpanetus »

neogodless wrote: post #167
arpanetus wrote: post #74

you can't just simply state that he's healthy without actually seeing his lifespan

Your assertion is that we have no way of assessing someone's health until they have died?

My assertion is that the every change we do towards things like that might involve million results and since it's not a simple logical statement it's rather hard to verify/validate the outcome in a short term due to the nature of dynamic/complex systems.

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#185

Post by mckravchyk »

ta1243 wrote: post #180
mckravchyk wrote: post #158

Earlier quoted context omitted.

Europe consists of many countries, and one of them is more than happy to take 9% / 5% of your income in exchange for one quite crappy public health care system - think whenever you need to see a specialist, you have to pay with your own money anyway (or wait months, years to get an appointment). If you want private health insurance, you have to fund the super expensive good for nothing public one anyway.

Which country? In the UK I earn a decent wage (well more than 80% of people in the UK), and last year I paid less than £3k / $3.5k for healthcare which covered me, my wife, my 2 children. It also covers my 70+ year old mother and my 90+ year old grandmother who don't earn enough to pay income tax. 80% of people pay far less than I do. What type of health insurance can I buy in the US for $290 a month to cover a famil…

Poland. This is a recent change introduced in 2022. It used to be some $100/mo

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#186

Post by mckravchyk »

gambiting wrote: post #164
mckravchyk wrote: post #158

Earlier quoted context omitted.

Europe consists of many countries, and one of them is more than happy to take 9% / 5% of your income in exchange for one quite crappy public health care system - think whenever you need to see a specialist, you have to pay with your own money anyway (or wait months, years to get an appointment). If you want private health insurance, you have to fund the super expensive good for nothing public one anyway.

I...don't see how that's relevant to what I said, but.....ok? I don't really disagree.

It's relevant because sure you can get a private insurance but you are already spending 5% of your income before tax on the public one and can't opt out from it. Like that's too much expenditure already to think about private health insurance.

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#187

Post by gambiting »

mckravchyk wrote: post #185
ta1243 wrote: post #180

Earlier quoted context omitted.

Which country? In the UK I earn a decent wage (well more than 80% of people in the UK), and last year I paid less than £3k / $3.5k for healthcare which covered me, my wife, my 2 children. It also covers my 70+ year old mother and my 90+ year old grandmother who don't earn enough to pay income tax. 80% of people pay far less than I do. What type of health insurance can I buy in the US for $290 a month to cover a famil…

Poland. This is a recent change introduced in 2022. It used to be some $100/mo

So I disagree with this statement where you said

>>you have to fund the super expensive good for nothing public one anyway.

My father had battled cancer in Polish oncology hospitals and has received nothing but top tier treatment wherever he went. What's more, NFZ completely funded the treatment with then-experimental Glivec for him, which was billed at around 200k zł/year(in US, a year of Glivec treatment is around $100k). In the course of 8 years of cancer he had to stop working due to the ilness, and yet was still fully covered and received the same treatment as always.

So no, it's not "expensive but good for nothing". It's not expensive compared to private treatment, and it's extremely good if you have a major ilness. I agree that routine treatment and appointments are in shambles, but that's universally true pretty much everywhere in Europe right now, the waiting times in countries with public healthcare systems have inflated into infinity due to a combination of factors. But even in Poland, I wouldn't dare to not pay into the system - it's too good not to pay for it(and I do, in fact, pay for it).

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#188

Post by gambiting »

HlessClaudesman wrote: post #143
gambiting wrote: post #39

Earlier quoted context omitted.

But....they can? You can give someone else the power to vote on your behalf if you cannot do it yourself. Unless you mean that if someone is literally unable to make any decisions by themselves their caretaker should be able to vote on their behalf how they think this person would vote? That doesn't strike me as a very reasonable system, you can't know how someone would vote unless they tell you, and to assume you ca…

Yeah I can see there are many sides, as a society we an interest in keeping elections fair, with one person one vote being a good maxim. But consider the case of a parent of a child with profound disabilities, that child is now an adult of voting age. Wouldn't a person with disabilities have an interest win voting out a candidate / party who constantly rails against providing funding for disabled services? Why should…

>>Wouldn't a person with disabilities have an interest win voting out a candidate / party who constantly rails against providing funding for disabled services?

Sure, but the point is, you don't know this - you can only reasonably guess. And to make a vote on behalf of another person, by effectively guessing what they might want, does not sit right with me.

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#189

Post by yCombLinks »

xen2xen1 wrote: post #84
wheelerof4te wrote: post #81

How exactly do you edit a gene? Like, it is very, very small thing. How do you even measure it, let alone replace it?

Viruses do that an astounding number of times a day, every day, don't they? And this treatment uses a virus.

A virus that we've modified to deliver the genetic payload we want inserted at a specific point in this child's DNA. It's not "give this child a random virus we have laying around, that'll fix her".

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#190

Post by lazyasciiart »

autoexec wrote: post #105
lazyasciiart wrote: post #51

Earlier quoted context omitted.

They really aren't that rare, and the reason we don't help all of them is not because of a utilitarian decision not to spend money on public healthcare. If you can get your humanitarianism to move that money from corn subsidies and fighter jets to health spending, more power to you.

> They really aren't that rare, 3,100 cases a year in all of Europe, and 3,600 cases a year in the entire US. That's pretty damn rare really. And it's a genetic condition, which means we should be working to eradicate the problem entirely by screening parents and gene editing ideally making it both rare and increasingly rare from here out.

It didn't occur to me that you were talking about this specific condition, rather than the set of conditions that will be plausibly treated this way.
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