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FDA approves a CRISPR-based medicine for treatment of sickle cell disease

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Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#201

Post by quickthrower2 »

eszed wrote: post #123
CobrastanJorji wrote: post #119

Earlier quoted context omitted.

It's a recessive/heterozygous thing. If you get the gene from neither parent, you're vulnerable to malaria. If you get the gene from either parent, you're immune to malaria and don't get sickle cell. If you get the gene from both parents, you get sickle cell. A hypothetical future person who's going to be born in an area with a lot of malaria would really want exactly one parent with sickle cell and one parent lackin…

Can they do sperm (or egg) selection to change those odds for IVF?

Or add the DNA tests to dating apps.

Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#202

Post by quickthrower2 »

seydor wrote: post #113
SamBam wrote: post #21

> Vertex set the price of Casgevy at $2.2 million > Patients must spend weeks, even months, in the hospital before and after the therapy is administered. Yoiks. So how many actual people are going to be able to get this treatment?

i always find those prices unbelievable. What is the actual cost of all the expense and workhours for making the treatment

Throw that stone in the SaaS glass house. $100/m for 10c of compute and $1 of a devs time.

Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#203

Post by quickthrower2 »

pknerd wrote: post #33

I wonder whether in the future doctors have a visual designer(WYSIWYG) similar what programmers have in the form of Visual Basic/QT to alter DNA. It'd be pretty interesting if it happens

I wonder if they will have a natural language chat interface

Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#204

Post by JumpCrisscross »

joshuamcginnis wrote: post #46

Here's basically how the process works: * Harvest stem cells from the patient. * Prepare a DNA plasmid with the Cas9 gene, guide RNA for the desired genetic modification, and an antibiotic resistance gene. * Electroporate the plasmid into the harvested stem cells. Grow the electroporated stem cells in antibiotic-containing nutrient media. Only cells with the plasmid (and thus antibiotic resistance) survive. * Expand…

Do you know why they're having the marrow synthesise fetal hemoglobin versus hemoglobin A(2)? (Is it simply because HbF is one molecule while HbA and HbA2 are two?)

Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#206

Post by fasteo »

Every time I read[1] about CRISPR there seem to be concerns about off-target editing, that might cause all kinds of trouble. Is this a solved issue ?

Note: I am not a doctor, but I do have a genetic condition that might benefit from CRISPR therapies, hence my interest.

[1] https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10034092/#:~:te....

Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#207

Post by imtringued »

freedomben wrote: post #158
Modified3019 wrote: post #105

Earlier quoted context omitted.

Maybe they are referring to the small percentage of people are sensitive to the whole family (potatoes, tomatoes, peppers, tobacco, etc) and find any exposure produces inflammation/digestion issues.

yes it's both actually! Potatoes are wildly toxic to humans when they have any green on them, and that can actually happen in the refrigerator if left to long and then consumed. They have the capacity to really F us up. Editing those strikes me as like threading a needle between hair triggers. You don't want to miss your target. But also yes exactly, the whole family can cause inflammation and difficulties in people…

Don't worry, once they figured it out they are going to put this stuff in every food and you won't be able to eat anything at the supermarket anymore.

Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#208

Post by imtringued »

w0mbat wrote: post #44

The gene that causes sickle cell anaemia actually provides partial immunity to malaria, which is why this gene has not been bred out of the population over time.

That is true but even the unreliable malaria vaccines that are out there are more effective and reliable against malaria than sickle blood cells.

Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#209

Post by refactor_master »

krapp wrote: post #110
refactor_master wrote: post #107

Earlier quoted context omitted.

Actually, utilitarianism and capitalism have been the strongest growth factors for human prosperity and welfare since forever. Many societies with excessively strong opinions on morals however are literally living in huts.

>Many societies with excessively strong opinions on morals however are literally living in huts. Setting aside that the US government is deeply influenced by Christian conservatism and the culture by Puritan ideals, to the point that no American President can be elected without vocally professing faith in God, Christ, or being seen with a Bible in hand, and thus is the most moralizing culture within Western civilizat…

Here's a graph showing the negative correlation between "hut-dwelling" and "utilitarianism":

https://www.pewresearch.org/short-reads/2019/05/01/with-high...

The US can be said to be both. E.g. you won't find many "huts" along the northeastern coast, but search and you'll find them elsewhere:

https://en.wikipedia.org/wiki/List_of_U.S._states_and_territ...

I'll leave it as an exercise for the reader to determine where the most "huts" are found.

So it's flat out wrong to claim that some sort of perceived moral bankruptcy with regards to the value of human life has left our society in the stone ages, when all evidence points to the contrary.

Under utilitarian capitalism people are expendable, but in the meantime they are less likely to dwell in huts than morally superior societies.

Note that I've blatantly equated religion with moral here.

Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#210

Post by imtringued »

ponector wrote: post #37
ericmay wrote: post #29

Earlier quoted context omitted.

This is Day 1 so the price and how well it works today is almost certainly the worst it will ever be. Insurance will likely cover the cost. It's a very bad, painful, and outright deadly genetic mutation and $2.2 million is practically nothing compared to doubling someone's lifespan or giving them an extra 10 years. More info I found relevant regarding cost for typical treatment and out of pocket estimated costs: http…

I don't agree that it is nothing. 2 millions, if applied properly, could do good for many people. Take ten children from poverty, give ten children chance to get a good education, etc. There is always a some kind of moral dilemma: should you spent millions to try to extend extremely I'll person or help with that money to some healthy poor children?

I'm sorry but the "cure" for that requires political change involving the stepping over of some people's corpses. You can only do that once people are sufficiently angry.

Healthcare? People pay to be and stay healthy. The money was earmarked specifically for this purpose. Also, in the long run, you will be able to cure diseases even those "healthy" children have.

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