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Posted On: 07/05/2022 12:44:26 AM
Post# of 153822
No, only one daughter cell would get the DNA episome. Translation of the episome (production of LRM) may wash out over time, as gene therapy does not last forever, at least not in the liver for Hemophilia A.
What would be the advantage of LRM gene therapy to block CCR5 versus gene Knockout to eliminate a functional CCR5 receptor? Sangamon and others gave tried CCR5 KO without much success, but it's possible they couldn't transfect enough autologous T Cells or HSPCs to evoke a cure. Then there are the Chinese gene edited embryos for CCR5 KO that seemed like it didn't take too well.
What would be the advantage of LRM gene therapy to block CCR5 versus gene Knockout to eliminate a functional CCR5 receptor? Sangamon and others gave tried CCR5 KO without much success, but it's possible they couldn't transfect enough autologous T Cells or HSPCs to evoke a cure. Then there are the Chinese gene edited embryos for CCR5 KO that seemed like it didn't take too well.
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