4 ms·
First you need a gene therapy delivery system that doesn't produce any off-target mutations at all, ever.
by roywiggins 10mo ago
First 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.