Question

What are the two main strategies of somatic cell gene therapy?

Answer 1

The two main strategies of somatic cell gene therapy are ex vivo gene therapy and in vivo gene therapy.

Step-by-step explanation:

The two main strategies of somatic cell gene therapy are:

1. Ex vivo gene therapy: This strategy involves collecting somatic cells from an affected individual, growing them in culture, and transfected them with retroviral cloning vectors containing the remedial gene construct. The transfected cells are then selected and transplanted back into the patient. Examples of diseases treated with ex vivo gene therapy include gaucher disease, sickle cell anemia, and thalassemia.
2. In vivo gene therapy: In this strategy, the remedial gene is directly delivered into the cells of a specific tissue in the patient using retroviral vectors or plasmid DNA constructs.

Both strategies use a vector, which is a carrier molecule for the gene. The vector helps incorporate the desired gene into the patient's DNA. Usually, the vector is modified viral DNA with the viral genes removed.