MaryBall

Because this therapy edits the stem cells that are found in the bone marrow, it essentially means that the disease is being fixed at the source. Bone marrow stem cells eventually turn into blood cells and immune cells. So if 100% of the cells have been edited, then the resulting cells they turn into will be the edited type.

Yes, you’re right! A lentiviral vector is used to insert the working gene into the patient’s stem cells. Obviously this is a non-pathogenic version of the virus, so it’s ability to make you ill has been essentially “switched off”. However the ability for the virus to insert genetic information into the DNA of cells remains.