Ask HN: Why are single-gene diseases not cured yet?

4 pointsnycdatasci2 comments

I'm specifically interested in SDS, which is a rare genetic disease that impacts blood production. It seems like numerous viable options exist for the modification of hematopoietic stem cell DNA, which I believe would partially cure the disease. Is this not just a matter of extraction (marrow) -> isolation -> modification -> confirmation -> implantation? What is holding us back?

Some references: * DIY genetic engineering kit: http://www.the-odin.com/genetic-engineering-home-lab-kit/

* Order custom CRISPR lentivirus vectors here: https://crispr.sigmainformatics.com/templates/CRISPR%20Custom%20Editable%20PDF_v8_savable.pdf

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