Question

Which is correct regarding ex vivo gene therapy?

• A. Therapeutic Genes are transferred to germ line cells
• B. Cells are modified outside body and transferred back to the body
• C. Genes are changed in cells when cells are inside the body
• D. Luxturna is an example of ex vivo gene therapy

Hint:

• Ex vivo Gene Therapy Cells removed from patient \(\rightarrow\) Genetically modified in lab \(\rightarrow\) Modified cells returned to patient. (Ex = Outside)
• In vivo Gene Therapy Therapeutic gene delivered directly into patient's body. (In = Inside)
• Somatic Gene Therapy Targets non-reproductive cells. Changes are not heritable. (Most current gene therapies).
• Germline Gene Therapy Targets reproductive cells (sperm, egg, embryo). Changes are heritable. Raises significant ethical concerns.

Answer 1

The Correct Option is B

Ex vivo gene therapy involves removing cells from a patient's body, modifying them outside the body to correct a genetic defect, and then reintroducing the modified cells back into the patient. This method is commonly used because it allows precise control over the genetic modification process in a laboratory setting before the cells are transplanted back into the patient. Let's examine the options:

• Therapeutic Genes are transferred to germ line cells: This is not correct for ex vivo gene therapy. This process involves somatic cells, not germ line cells.
• Cells are modified outside body and transferred back to the body: This is the correct description of ex vivo gene therapy. The cells are altered in a controlled environment outside the body.
• Genes are changed in cells when cells are inside the body: This describes in vivo gene therapy, not ex vivo.
• Luxturna is an example of ex vivo gene therapy: Luxturna is an example of in vivo gene therapy, where the therapeutic genes are directly delivered into the body.

Thus, the statement "Cells are modified outside body and transferred back to the body" is correct regarding ex vivo gene therapy.