Question

Why are diseases of the blood more likely targets for treatment by gene therapy than are many other genetic diseases?

1) Because blood cells are highly susceptible to viruses which are commonly used as vehicles to deliver therapeutic DNA inside the cell.
2) Because genetically modified blood cells cannot evoke the immune response.
3) Because the bone marrow cells that give rise to blood cells can be isolated, cultured and manipulated in vitro.
4) Because blood cells have the shortest life cycle.

Answer 1

Blood diseases are targeted by gene therapy due to the ability to manipulate bone marrow cells ex vivo, which are then reinserted into the body to produce healthy blood cells.

Step-by-step explanation:

Diseases of the blood are more likely targets for treatment by gene therapy than many other genetic diseases primarily because the bone marrow cells that give rise to blood cells can be isolated, cultured, and manipulated in vitro. This approach is known as ex vivo gene therapy, where somatic cells from an individual are collected, modified outside the body, and then reintroduced into the patient. Diseases such as sickle cell anemia and thalassemia have been treated using this technique. Viral vectors, like modified adenoviruses, are often used to deliver the therapeutic genes into the bone marrow cells. Although gene therapy using viruses as carriers is still experimental and there are associated risks, such as immune responses and potential unintentional gene disruptions, it holds promise for the treatment of many genetic diseases.