An overview of Gene Therapy

1. GENE THERAPY

2. INTRODUCTION
• Gene therapy is the use of DNA as a drug to treat
disease by delivering therapeutic DNA into a
patient‘s cells.
• Genetic modification of cells in a patient is done.
• The most common form of gene therapy involves
using DNA that encodes a functional, therapeutic
gene to replace a mutated gene.

3. HISTORY
• Gene therapy was first conceptualized
in 1972 and was performed on various
animals.
• The first FDA-approved gene therapy
experiment in the United States
occurred in 1990, when Ashanti DeSilva
was treated for ADA-SCID.
• In 2012, Glybera became the first gene
therapy treatment to be approved for
clinical use in either Europe or the
United States.
FIGURE----------- Ashanti Desilva

4. TARGET DISEASE FOR GENE THERAPY
• Gene therapy is provided for diseases which do not
have other alternative treatment. For example,
 Multiple myeloma
 Aids
 Immune system disorder (SCID)
 Huntington’s chorea
 Cystic fibrosis
 Haemophilia

5. TYPES OF GENE THERAPY
• SOMATIC GENE THERAPY:-
In somatic gene therapy, the therapeutic genes are
transferred into the somatic cells (non sex-cells), or
body, of a patient.
• GERMLINE GENE THERAPY:-
In germline gene therapy, germ cells (sperm or
eggs) are modified by the introduction of functional
genes, which are integrated into their genomes.

6. VECTORS IN GENE THERAPY
• The two major classes of methods are those that use recombinant
viruses (viral vectors) and those that use naked DNA or DNA
complexes (non-viral methods).
• VIRAL VECTORS:- A number of viruses have been used for human
gene therapy, which includes,
 Retrovirus
Adenovirus
Lentivirus
Herpes simplex virus
Adeno- associated virus

7. VECTORS IN GENE THERAPY
• NON-VIRAL METHODS:-
There are several methods for non-viral
gene therapy, which includes ,
 Electroporation
 Gene gun
 Lipofection
 Inorganic nanoparticles
 Sonoporation
 Microinjection

8. METHODS FOR GENE THERAPY
• INVIVO GENE THERAPY :-
Gene is directly injected into the cells of
the patient and cells are genetically modified insitu.
• EXVIVO GENE THERAPY:-
Cells are removed and are genetically
modified outside the body.

9. FIGURE:- IN-VIVO & EX-VIVO GENE THERAPY
METHODS OF GENE THERAPY

10. USES OF GENE THERAPY
• Gene Doping
• Human Genetic Engineering
Used to change physical appearance,
metabolism, and even improve physical
capabilities and mental faculties like memory
and intelligence