4 ms·
Eh, while that's true for many things, there are plenty of genetic diseases for which it is not ("diseases" or whatever you might call the human lack of vitamin
by colechristensen 11mo ago
Eh, while that's true for many things, there are plenty of genetic diseases for which it is not ("diseases" or whatever you might call the human lack of vitamin C synthesis)
In this case the gene encoding L-gulonolactone_oxidase is broken, and that's the last step in the process. That gene catalyzes something into a substance which decays into vitamin C.
- roywiggins 11mo agoFirst you need a gene therapy delivery system that doesn't produce any off-target mutations at all, ever.
- colechristensen 11mo agoExtract tissue from patient, build a cell line, CRISPR in vitro, build a cell line, sequence to verify. Use verified cell line to build pseudo-organs or to inject cells or stem cells. ex vivo gene therapy.
- roywiggins 11mo agoCells don't survive sequencing, any that you do implant have not been sequenced. At best you can get some confidence that the error rate is small.
- colechristensen 11mo agoThis is why I said build a cell _line_, i.e. cells that all come from a single parent cell. Clones. Make monoclonal stem cell lines, use CRISPR on them, make a NEW monoclonal cell line post-CRISPR and pull some cells to validate success or failure.