Does anyone who's more familiar with exactly how this works know if this could be applied or potentially applied to thalassemia? From what I understand those are also related to genetically-driven misshapen red blood cells.
CRISPR Therapeutics also has a therapy for Beta Thalassemia in human trials that is up for approval in February 2024.
“Editing Humanity” by Kevin Davies covers the history and near future of CRISPR and includes a great chapter on describing both of these therapies.
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.
> which is why this gene has not been bred out of the population over time. Is that why? Or is it just the people with it aren’t sick enough to die before procreating?
Africa wasn't colonized by Europe until vaccines and treatments were invented because of malaria and other tropical diseases. Quinine was one of the last ingredients needed to conquer Africa.
> 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
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…
is crispr a big improvement here over AAVs or zinc fingies?
AAV or adeno-associated virus is a delivery method for getting cas9 mRNA (the code that says, make cas9 protein and do gene editing). Zinc finger nucleases are a similar class of dna editing proteins.
In this specific experiment they chose to transfect cells with plasmid directly rather than transduce with virus.
Lyfgenia’s approval came with a black box warning about the possibility that patients who receive the therapy might later develop blood cancer and should be monitored for that risk. Two patients in trials of the drug died of blood cancers, and studies concluded that the cancers were caused by the chemotherapy conditioning regimen for the treatment, not Lyfgenia itself.
>the cancers were caused by the chemotherapy conditioning regimen for the treatment, not Lyfgenia itself. I am certain some media group is going to conveniently leave this part out of the title of their article, and surely no one is gonna waste time reading the actual article and the rumors will take off.
The article says "patients must undergo a preparatory treatment with a chemotherapy drug to remove any native stem cells that might remain in their bone marrow." It doesn't make much difference to the patient if it's the Lyfgenia itself or the chemo drug, if the chemo drug is a requirement. Right?
Ohalo (the company Dave Friedberg is now CEO of) recently got approval for a potato edited by CRISPR: > Ohalo had two RSRs under consideration this year for its potato, one which focuses on higher concentrations of beta carotene – enhancing the overall health and nutrition value of the potato – and another which results in reduced glucose and fructose content in the potato, which, according to Ohalo, will reduce the…
>and another which results in reduced glucose and fructose content in the potato, That's pretty amazing. Imagine if we can change apples to produce Aspartame instead of sugars!
I don’t see how a plant is supposed to metabolize the aspartame for energy?
Collectively paying for rare but expensive treatments is literally the problem that insurance solves. This isn't wildly out of the expected range for this sort of thing. And it will surely get cheaper as it evolves.
Since the collective probability of rare, but expensive health issues is basically 100%, I would describe it less as insurance and more as wealth redistribution. Hence the (typical) requirement to purchase insurance and lack of ability to price it based on risk. Of course, insurance and taxation can be viewed as similar things anyway, but it is different from things like term life insurance or motor vehicle insurance…
> the collective probability of rare, but expensive health issues is basically 100%
Not really, no. Most people will die of something expensive, but not $2M expensive. A quick google says that per-capita lifetime health care expediture is ~$300k.
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?
Such a crass statement. What if you're the patient? Would you spend 2 million to live 30-40 more years? It's so easy to step back and weight the lives of other as if you're making the decision for others.
Crass, sure.
Not sure it's really easier though, economics and emotional affect are often at odds. Ask people if a hospital administrator should spend 100k on either a single liver transplant for an 11 year old girl, or spread over 100 less expensive life saving interventions for 50 year olds, most people will say save the girl and demand the administrator be fired for even needing to think about it.
(Half remembered but apparently real scenario, though I'm not sure where from)
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.
> which is why this gene has not been bred out of the population over time. Is that why? Or is it just the people with it aren’t sick enough to die before procreating?
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 lacking the gene completely to guarantee the best personal outcome, or they'd want exactly one heterozygous parent (for a 50% chance of being immune to malaria with no downside), or they might settle for the gamble of two heterozygous parents (50% chance of immunity, 25% chance of sickle cell).
Ohalo (the company Dave Friedberg is now CEO of) recently got approval for a potato edited by CRISPR: > Ohalo had two RSRs under consideration this year for its potato, one which focuses on higher concentrations of beta carotene – enhancing the overall health and nutrition value of the potato – and another which results in reduced glucose and fructose content in the potato, which, according to Ohalo, will reduce the…
>Of the ten plant-based proteins included in the current analysis, potato protein is the only protein source containing the WHO/FAO/UNU requirements for all essential amino acids. Thus, when consuming potato protein as the only dietary protein source at the recommended adult protein intake level of 0.66 g/kg/day, sufficient amounts of all essential amino acids should be consumed. It remains to be investigated whether the ingestion of a single meal-like amount of potato protein has the capacity to stimulate muscle protein synthesis.
Anyone know about vegan protein profiles and best ones now?