Three people with inherited diseases successfully treated with CRISPR

It&#x27;s interesting that they did <i>not</i> just undo the inherited disease. I assumed that, especially with Sickle Cell where we have a good understanding of how it works, they would go into Chromosome 11 and put it back how it &quot;should&quot; be with CRISPR. But instead they apply a workaround, ensuring continued fetal haemoglobin production.<p>The article does not mention whether that&#x27;s because putting Chromosome 11 back with CRISPR is harder, or whether for some reason that wouldn&#x27;t fix the problem.

This is such mind-blowing success, it makes me contemplate switching fields. Is it reasonably feasible to switch from IT to Biotech&#x2F;Medical Research in the mid thirties?<p>I had a friend with Fukutin-related limb-girdle muscular dystrophy R13 and am wondering if CRISPR could be the solution for her.

&gt;The remaining bone marrow cells are killed by chemotherapy, then replaced by the edited cells.<p>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?

Smalll thing, Beta thalassaemia isn’t a disease — as far as I know. It’s a disorder or a syndrome.<p>Definition of disease: Resulting from a pathophysiological response to external or internal factors.<p>Definition of disorder: A disruption of the disease to the normal or regular functions in the body or a part of the body.<p>Edit: I have it myself (and my kids do too), and I often correct people on this. Since I rather have a disorder than a disease:)

Note that this only edits the bone marrow cells, it is totally different from the scandalous experiment on gene editing in human embryos: <a href="https:&#x2F;&#x2F;www.livescience.com&#x2F;64166-first-genetically-modified-babies-risks.html" rel="nofollow">https:&#x2F;&#x2F;www.livescience.com&#x2F;64166-first-genetically-modified...</a>

Fetal hemoglobin binds stronger to oxygen than normal hemoglobin, so that a fetus can &quot;steal&quot; oxygen from its mother&#x27;s blood. But if the mother has been crispred to also have fetal hemoglobin, then this won&#x27;t work right? Meaning it&#x27;s a male only treatment?

I like how evolution if you can call it that figured out first that fetal haemoglobin mitigated the problem.<p>The fix used CRISPR (and a gene engine?) to implement the fix on other people.<p>It&#x27;s like version 2 of what human bodies figured out first naturally.<p>This sounds like something that my dad or other people with COPD&#x2F;IPF could use. If fetal haemoglobin could grab more oxygen it would benefit people with low lung function. Sort of like Star Trek &quot;Trioxin&quot; they loved to use.

I wonder since fetal hemoglobin has stronger binding with oxygen which means the blood can oxygenate faster if this can be used for other issues as well like reduced lung capacity and performance enhancement.

I&#x27;m hoping that other then treating diseases this thing can aid with enhancement.

But will the drug approval agencies allow genetically engineered therapies for anything disruptive? For example, they&#x27;ve been blocking the caries vaccine for decades.