" By way of non-limiting example, a wild type gene, e.g. encoding at least one globin (e.g., a globin, .gamma. globin, .beta. globin and combinations thereof), may be inserted into a cell (e.g., into an endogenous BCL11A enhancer sequence using one or more nucleases as described herein) to provide the globin proteins deficient or lacking in the cell and thereby treat a genetic disease, e.g., a hemoglobinopathy, caused by faulty globin expression."
Interesting that they are considering inserting the normal beta globulin gene or fetal gene at the site of the BCL11A enhancer knockout.
This would not only knock out the BCLA11 enhancer but would also produce the beta/fetal hemoglobin also..
2 birds, one stone.
They probably aren't just inserting the new healthy gene at the normal beta globin site for some reason..no one else is doing that either..
Possibly gene is too large to completely cut out and then insert entire new gene?
Problems with knocking out endogenous beta globin gene with the deleted area causing problems with cell development?
Anyway, no one else doing it either, so there must be an issue.