Question

Write a note on uses of somatic cell gene therapy.

Answer 1

Somatic cell gene therapy:

1. In this type the gene is introduced only in somatic cells like bone marrow cells, hepatic cells, fibroblasts endothelium and pulmonary epithelial cells, central nervous system, endocrine cells and smooth muscle cells of blood vessel walls.
2. Somatic cell gene therapy is the only feasible option and the clinical trials have already employed for the treatment of acquired disorders such as cancer and rheumatoid arthritis and blood disorders including SCID, Gaucher’s disease, familial hypercholesterolemia, haemophilia, phenylketonuria, cystic fibrosis, sickle cell anaemia, Duchenne muscular dystrophy, emphysema, thalassemia etc.