Gene Therapy vs Sickle Cell Anemia

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Ares
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Gene Therapy vs Sickle Cell Anemia

Post by Ares » Mon Mar 11, 2019 3:22 am

I saw a 60 Minutes episode on Could Gene Therapy Cure Sickle Cell Anemia , and thought "This is the kind of science I love", the kind of thing that seems like science fiction brought to life.

Essentially, there are certain genetic diseases that scientists know are caused basically by one "flipped" strand of DNA. Through use of a modified HIV virus and the patients own stem cells, they've apparently found a way to correctly flip the DNA into the right position. So far the people in the trials are effectively cured of the genetic defect.

Naturally, this needs a lot more time and study to make sure this is an actual cure and not simply a temporary fix, but if it does prove to be a permanent solution, this could be a huge game changer. The guy mentions there are several thousand genetic disorders like this that they know the exact strand that needs fixing in this manner. Being able to permanently cure all of them would be an incredible leap forward in medicine.

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Re: Gene Therapy vs Sickle Cell Anemia

Post by Ken » Mon Mar 11, 2019 1:09 pm

Rewriting DNA sounds scary. Curing genetic disorders, if it works, would be great. But the possibilities of the damage that could be wrought if misused seem tremendous.
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Re: Gene Therapy vs Sickle Cell Anemia

Post by Ares » Mon Mar 11, 2019 2:40 pm

It's definitely got potential for abuse, but that just makes it like any other emerging technology. It'll require a lot of testing (for instance, to make sure the children people treated in this manner don't pass on some new kind of genetic abnormality to their children), and it'll require laws to prevent things like potentially using this to custom design babies or try to "fix" people with traits they don't like. But at the end of the day, the potential to cure diseases like this and future advancements are beneficial enough to be worth the risk.

I've read enough science fiction to know where this could go, and where human nature could take it, but overall I see this as something positive.

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Re: Gene Therapy vs Sickle Cell Anemia

Post by Woodclaw » Mon Mar 11, 2019 10:42 pm

Interesting little factoid, sickle cell anemia has a "positive" side-effect: it makes people immune to malaria.
During World War 2, the only troops that registered almost zero malaria losses came from southern Italy (particulary Puglia and Calabria), where the sickle cell anemia was extremely common among the male population. Post-war studies linked the two facts and demonstrated that the malaria virus can't infect sickle red blood cells the way they do with normal ones.
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Ares
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Re: Gene Therapy vs Sickle Cell Anemia

Post by Ares » Mon Mar 11, 2019 11:07 pm

It's partially why sickle cell anemia is so wide-spread in Africa. Though it's a shame that being immune to one disease comes with crippling bouts of pain.

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Re: Gene Therapy vs Sickle Cell Anemia

Post by catsi563 » Fri Mar 15, 2019 7:28 pm

Im looking forward to the possibilities of Stemcells being used to cure Alzheimer's Dementia and possibly other neurological disorders like those caused by a stroke. bit of a personal stake in that last one. the hope and possibilites that those above and others such as Diabetes and cancers can be cured with whats in our own body if we can just get the religious nonsense out of the way of actual science is profound
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Re: Gene Therapy vs Sickle Cell Anemia

Post by Prof Weird » Tue Mar 19, 2019 6:42 am

Since the researchers are altering stem cells, that would cure the person they're injected into. They will not be able to pass on the changes to their children though, since their germline is not altered.

Sickle cell anemia is an example of heterozygous advantage - in areas with malaria, people with two copies of regular hemoglobin die from malaria, while those with two doses of sickle cell have health trouble. Heterozygotes (have one copy of each gene) have the greatest benefit; IIRC, the cells only sickle when infected with malaria parasites or under rather low oxygen titers.

HIV virus can deliver DNA to the cells, where I'd guess repair mechanisms will splice it into the genome (hopefully the correct spot; some techniques just allow the DNA to insert anywhere in the genome). Depends on whether the researchers are relying on homologous recombination or safe harbor targeting.

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