Basics overview of gene therapy

1. GENE THERAPY
Subh Naman
M Pharm(Pharmaceutics)
DPST,MRSPTU

2. WHAT IS GENE THERAPY?
• An experimental technique for correcting defective genes
that are responsible for disease development.
• The most common form of gene therapy involves
inserting a normal gene to replace an abnormal gene
• Other approaches used:
-Replacing a mutated gene that causes disease with
healthy copy of the gene.
-Inactivating, or “knocking out,” a mutated gene that is
functioning improperly.

3. Candidate Disease for Gene Therapy
1. Severe Combined Adenosine deaminase Bone marrow
immunodeficiency(SCID/ADA) cell or T-cell
2. Hemophilia
3. Cystic fibrosis Loss of CFTR gene Airways in lung
4. Hemoglobinopathy Thalessimia structural defect bone
marrow Sickel cell anemia of alpha/beta- globin gene
5. Parkinson's disease
6. Cancer
7. HIV

4. HISTORY AND DEVELOPMENT
OF GENE THERAPY
• 1960: The concepts of Gene Therapy was introduced.
• 1990:
The first approved gene therapy case at the National Institute
of Health, U.K. It was performed on a four year old girl named
Ashanti DaSilva. It was a treatment for a genetic defect that
left her with a.n immune system deficiency.
• 2006:
Scientists at the National Institutes of Health (Bethesda,
Maryland) have successfully treated metastatic melanoma in
two patients.

6. GERM LINE GENE THERAPY
• Result in permanent changes.
• Potential for offering a permanent therapeutic effect for all
who inherit the target gene.
• Possibility of eliminating some diseases from a particular
family.
• Therapeutic genes transferred into the germ cells.
• Eg. Genes introduced into eggs and sperms.

7. SOMATIC CELL GENE THERAPY
• Affects only the targeted cells in the patient, and is not
passed to future generations.
• Short-lived because the cells of most tissues ultimately die
and are replaced by new cells.
• Therapeutic genes transferred into the somatic cells.

10. EX-VIVO

27. THANK YOU