4 ms·
Ask HN: Why are single-gene diseases not cured yet?
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
- danieltillett 9y agoThe answer has three letters - FDA.
- nycdatasci 9y agoHopefully the FDA gets out of the way before a thriving black market emerges. After reading about your firm, it's clear that I'm taking some things for granted like the quality of a DNA sequence. A single error when validating a gene modification could be fatal. Thanks for doing your part to advance this tech!