GENE THERAPYBy,

D.AkshayaB.GOKULA PRIYA

SCHEMA GENE THERAPY-

DEFINITION HISTORY-GENE

THERAPY TECHNIQUES AND

TERMS TYPES OF GENE

THERAPY ADVANTAGES AND

DISADVANTAGES CONCLUSION

DEFINITION

Gene therapy is the use of DNA as a pharmaceutical agent to treat disease. 

BASICS… DNA: Genes are composed of DNA, a molecule in the

memorable shape of a double helix, a spiral ladder. Each rung of the spiral ladder consists of two paired chemicals called bases. There are four types of bases. They are adenine (A), thymine (T), cytosine (C), and guanine (G). As indicated, each base is symbolized by the first letter of its name: A, T, C, and G. Certain bases always pair together (AT and GC). Different sequences of base pairs form coded messages.

The gene: A gene is a sequence (a string) of bases. It is made up of combinations of A, T, C, and G. These unique combinations determine the gene's function, much as letters join together to form words. Each person has thousands of genes -- billions of base pairs of DNA or bits of information repeated in the nuclei of human cells --which determine individual characteristics (genetic traits).

HISTORY… The concepts of gene therapy arose

initially during the 1960s and early 1970s ….

With the arrival of recombinant DNA techniques, cloned genes became available

Efficient retroviral vectors and other gene transfer methods have permitted convincing demonstrations of efficient phenotype correction in vitroand in vivo, now making gene therapy a broadly accepted approach to therapy

TECHINQUE…….PICTORIAL REPRESENTATION..

STEPS INVOLVED.. Cells from the patient’s blood or bone marrow are removed

(usually cells containing defective genes) The cells are exposed to viruses,carrying the desired

genes… Then,the cells are grown in the laboratory and then

injected into the patient’s body.

EXVIVO GENE THERAPY

METHODS OF GENE TRANSFER Viral method-uses

viral vectors for gene transfer…

Method: Viruses are first disabled to replicate themselves and are exposed to the cells

Drawback:possiblities for vial multiplication-leads to new diseases..

NON-VIRAL METHODS

Electroporation-electric field

Sonoporation-ultasonic frequencies

Gene gun-bombardment

Magnetofection-magnetic field

VIRAL VECTOR!!!!

TYPES OF GENE THERAPY.. SOMATIC -gene

therapy

Gene transfer to somatic cells…

The effect is not hereditary i.e confined to the individual

GERM LINE-gene therapy

Gene transfer to germ cells viz..sperm or egg

The effect is hereditary

DISEASES CURED…Genetic disorders Severe Combined

Immune Deficiency Chronic

Granulomatus Disorder

Hemophilia

Acquired diseases Cancer Neurodegenerative

diseases HIV

PROS AND CONS…Advantages

arrests some diseases gives patients new genes, so

they don't have the disease and the disease doesn't come back

provides as a way of treatment when in the past the disease would have been incurable

there have been several medical breakthroughs

some people consider it a great advancement for mankind

DisadvantagesIn the past, gene therapy has cured some diseases, but caused others

Its inconsistent Its a fairly new method, so

there are still some glitches to it that scientists are still figuring out.

some people are against the ethics of it: some say its "playing God"

the patient may have to undergo several therapy treatments

CONCLUSION… GENE-THERAPY is an upcoming field and

in the future i.e within a decade its going to be the cure for every genetic disease and the other methods would go a cliche!!

THANK YOU!!!