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by colechristensen 192 days ago
Extract 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.

1 comments

Cells 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.
This 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.