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by rotexo 2841 days ago
Gene therapy has had some recent successes [0], but it is worth noting that many of the ongoing and recent clinical gene therapy trials don’t necessarily involve genome editing: go to clinicaltrials.gov, search ‘gene therapy’ and get 3741 results. Search ‘CRISPR’ or ‘TALEN’ and get ~21 results.

Edit: I should say that most of the gene therapy clinical trials don’t involve genome editing yet. Though it seems to me that, at least for some diseases, viral vectors that deliver DNA to cells without integrating DNA into the host cell genome (where the dna instead persists inside the cells episomally, and is maybe lost from the cells after some number of divisions) will have advantages over integrating vectors/genome editing-based approaches. That way, you don’t have to worry about the off-target effects of genome editing, and you have a treatment that doesn’t result in permanent changes to the genome.

[0] https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4879992/