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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.


First you need a gene therapy delivery system that doesn't produce any off-target mutations at all, ever.
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.

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.

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