Fetal hemoglobin binds stronger to oxygen than normal hemoglobin, so that a fetus can "steal" oxygen from its mother's blood. But if the mother has been crispred to also have fetal hemoglobin, then this won't work right? Meaning it's a male only treatment?
Three people with inherited diseases successfully treated with CRISPR
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Re: Three people with inherited diseases successfully treated with CRISPR
#32Earlier quoted context omitted.
Interesting, can you give a bit more details what exactly his path was?
comp sci in prestigious european uni. Moved to us in early 2000s. Big nationwide datacenter stuff. Decided to become doctor. Did doctory stuff and floated around medical for a while before switching to psyc. Currently forensic psych for infamous US prison. Found that rather than patient hop in regular psyc practice, or drug up people in asylums, with trial cases spanning years you can really deep dive a person and do…
Did he support a family during his transition or was he still single?
Was money a problem in general along the way?
Re: Three people with inherited diseases successfully treated with CRISPR
#33Fetal hemoglobin binds stronger to oxygen than normal hemoglobin, so that a fetus can "steal" oxygen from its mother's blood. But if the mother has been crispred to also have fetal hemoglobin, then this won't work right? Meaning it's a male only treatment?
... Or all the women who do not want any (more) biological children or find this side effect better than the disease? Making babies is not a raison d'etre in modern civilization.
Re: Three people with inherited diseases successfully treated with CRISPR
#34Fetal hemoglobin binds stronger to oxygen than normal hemoglobin, so that a fetus can "steal" oxygen from its mother's blood. But if the mother has been crispred to also have fetal hemoglobin, then this won't work right? Meaning it's a male only treatment?
Means that it is a treatment for anyone not able to have children, which is about half of females (post-menopausal, plus other infertilities).
Re: Three people with inherited diseases successfully treated with CRISPR
#35This is such mind-blowing success, it makes me contemplate switching fields. Is it reasonably feasible to switch from IT to Biotech/Medical Research in the mid thirties? I had a friend with Fukutin-related limb-girdle muscular dystrophy R13 and am wondering if CRISPR could be the solution for her.
It’s worth knowing there are very few diseases that can be cured with singular gene edits. Many diseases involve multiple genes, cascades of gene expression, or complex physiological pathways. Oh, and not to mention epigenetics and environmental factors.
The strategy they used speaks exactly to your point. The diseases were not "cured" in the sense that these people were brought back to a generic baseline. That very well may have required a complex series of edits that are well beyond what is currently possible. Instead, they focused on a clever workaround that was simpler to implement, reactivating fetal hemoglobin. That's how they were able to cure two different diseases with the same gene edit.
So you have a much larger pool of genetic and non-genetic diseases that can be cured now. For example if someone had low lung function due to lung scarring, this might be an interesting treatment to look at.
Re: Three people with inherited diseases successfully treated with CRISPR
#36Fetal hemoglobin binds stronger to oxygen than normal hemoglobin, so that a fetus can "steal" oxygen from its mother's blood. But if the mother has been crispred to also have fetal hemoglobin, then this won't work right? Meaning it's a male only treatment?
Means that it is a treatment for anyone not able to have children, which is about half of females (post-menopausal, plus other infertilities).
Add to that all women willing to give up the ability to have children for the treatment, which might be sizeable
Re: Three people with inherited diseases successfully treated with CRISPR
#37>The remaining bone marrow cells are killed by chemotherapy, then replaced by the edited cells.
Interesting that they need to kill the remaining bone marrow cells. I wonder what happens when they skip that step. Do the modified cells still reproduce, resulting in a partially effective treatment? Are they targeted by the immune system and eliminated? Are they just out-reproduced by the normal cells and fade into irrelevance?
Re: Three people with inherited diseases successfully treated with CRISPR
#38Earlier quoted context omitted.
Means that it is a treatment for anyone not able to have children, which is about half of females (post-menopausal, plus other infertilities).
Add to that all women willing to give up the ability to have children for the treatment, which might be sizeable
Because of accidental pregnancies, there are some treatments denied to all fertile women regardless of choice. Ussually they are those that cause horrible birth defects (limb reductions etc) or make pregnancy deadly to the mother. Given the links between oxygen and injuries like FAS, this hemoglobin treatment may be one of that group.
Example: Acutane was once used to treat acne in teenagers. Many/most doctors didnt give it to teenage girls regardless of whether they were sexually active or on the pill. Mistakes happen. That drug would do horrible things to a fetus.
Re: Three people with inherited diseases successfully treated with CRISPR
#39>The remaining bone marrow cells are killed by chemotherapy, then replaced by the edited cells. Interesting that they need to kill the remaining bone marrow cells. I wonder what happens when they skip that step. Do the modified cells still reproduce, resulting in a partially effective treatment? Are they targeted by the immune system and eliminated? Are they just out-reproduced by the normal cells and fade into irrel…
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Re: Three people with inherited diseases successfully treated with CRISPR
#40I like how evolution if you can call it that figured out first that fetal haemoglobin mitigated the problem.
The fix used CRISPR (and a gene engine?) to implement the fix on other people.
It's like version 2 of what human bodies figured out first naturally.
This sounds like something that my dad or other people with COPD/IPF could use. If fetal haemoglobin could grab more oxygen it would benefit people with low lung function. Sort of like Star Trek "Trioxin" they loved to use.