The transduced cells are grown in culture, collected
in large amounts, and then introduced into the
patient at various intervals; with the hope that
the cells will be maintained and the disease that is being treated will be corrected.
Patients with genetic diseases that respond to bone
marrow transplantation are treated by ex vivo gene therapy.
Bone marrow transplantation acts as a therapeutic
procedure for different genetic diseases, bone marrow
contains totipotent embryonic stem cells at a
frequency of 10-4 to 10-5. These embryonic cells can
divide and differentiate into various important cell
types, including B and T lymphocytes, macrophages, Red blood cells, platelets and bone cells.
Ex vivo gene therapy is a procedure in which genetically engineered totipotent stem cells are transplanted into a patient, where they would provide the missing cell type or gene product.
In-vivo Gene therapy:
Gene therapy involves direct delivery of a corrected
gene into the cells of a particular tissue of patient.