It turns out that if you want to correct sickle cell disease, you (just) have to revert the single the bit of genetic instruction that permits hemoglobin protei

It turns out that if you want to correct sickle cell disease, you (just) have to revert the single the bit of genetic instruction that permits hemoglobin proteins to atomically interlock like legos. The aggregation only occurs when the protein structures are well-suited to aggregation. A single atomic 'feature' on the protein is the difference between a protein that aggregates easily and one that does not. Further, there is actually a separate mutation to the hemoglobin protein that actively disrupts the interlocking of even those hemoglobins that would otherwise do so. So the presence of an additional atomic feature or bump on the protein actually inhibits aggregation when intermixed with proteins that would otherwise aggregate. Companies [1] are right now developing gene therapies [2] that introduce that 'upgraded' version of hemoglobin in patients. The upgraded hemoglobin has a mutation from a threonine to a glutamine at position 87 of wild-type human hemoglobin: https://serotiny.bio/notes/proteins/hbb/ https://serotiny.bio/notes/proteins/hbb/ [1] https://www.bluebirdbio.com/our-focus/severe-diseases/ https://www.bluebirdbio.com/our-focus/severe-diseases/ [2] http://www.nejm.org/doi/full/10.1056/NEJMoa1609677?query=featured_home& http://www.nejm.org/doi/full/10.1056/NEJMoa1609677?query=fea...