Very dumb question, but how exactly does gene therapy work in vivo? Don't you have to modify every cell (or affected type of cell) with a new copy of the DNA? How does that actually.... happen?
> Don't you have to modify every cell (or affected type of cell) with a new copy of the DNA?
It depends on the specific disorder that’s being treated. For some of them, targeting a fraction of the affected cells is enough to get the desired effect.
If the affected protein is actively causing problems, then you'd need to get most of the cells. But if it's just inert and not doing its job then you only need to get enough cells to get the production of the right version up to a somewhat functional level.
It's like trying to persuade enough people (cell) to change their mind so a social revolution takes place (cure). The argument (gene vector) must be very popular.
It depends on the specific disorder that’s being treated. For some of them, targeting a fraction of the affected cells is enough to get the desired effect.