4 ms·
WRT genetic engineering, I believe the main barrier to these things is that our genes are quite multipurpose. You may turn on the ability to produce vitamin C,
by devmor 10mo ago
WRT genetic engineering, I believe the main barrier to these things is that our genes are quite multipurpose. You may turn on the ability to produce vitamin C, and that same sequence of genes could also turn your eyeballs into calcified lumps.
- colechristensen 10mo agoEh, 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 10mo agoFirst you need a gene therapy delivery system that doesn't produce any off-target mutations at all, ever.
- colechristensen 10mo 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 10mo 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 10mo 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.