Gene therapy

Is the process of inserting genes in to

cells to treat diseases

Approaches for gene

theraphy
Somatic cell gene therapy
Non-reproductive cells
Eg. Bone marrow cells, blood cells,
skin cells, intestinal cells
Germ cell gene therapy
Reproductive cells

Major strategies in gene

therapy
Gene augmentation therapy
DNA is inserted into genome to
replace the missing gene product
Gene inhibition therapy
The antisense gene inhibits the
expression of the dominant gene

Gene therapy stratagies

Types of gene therapy

Ex vivo
Transfer of genes in cultured cells
Use of the patients own cells for
culture and genetic correction, and
then their return back to the
patients

In vivo
Direct delivery of genes into the
cells

Ex vivo

In vivo gene therapy

Gene therapy

Sever combined immunodeficiency disease

Hemophilia B
Thalassemia
Sickle cell anemia
Head and neck cancer
Glioblastoma (brain),
ovarian
Renal cancer, colorectal cancer
Breast cancer
AIDS
Diabetes