Presented By: Disha Bedi Class XII B

GENE THERAPY

Do you know that a single gene alteration by the process of gene therapy can prevent hereditary cancers and can even cure them.

A single gene alteration by the process of gene therapy can cure disorders such as Parkinson disease, Huntington disease, blood abnormalities, hemophilia and immunological disorders.

A single gene alteration by the process of gene therapy can make the world free from HIV AIDS.

A single gene alteration by the process of gene therapy can allow people to have children where natural conception is impossible.

What is this GENE THERAPY about which the whole world is talking?

                                                                                

WHAT IS GENE THERAPY?

Gene therapy is a technique for Gene therapy is a technique for correctingcorrecting the the defective genesdefective genes responsible for developmentresponsible for development of of diseases.diseases.

GENE Genes, which are carried Genes, which are carried

on on chromosomeschromosomes, are the , are the basic physical and basic physical and functional units of functional units of heredity. When heredity. When genes genes are alteredare altered so that the so that the encoded proteins are encoded proteins are unable to carry out their unable to carry out their normal functions, normal functions, genetic disordersgenetic disorders can can result.result.

WAYS TO GENE THERAPY

Researchers may use one of Researchers may use one of several approachesseveral approaches for for correcting faulty genes:correcting faulty genes:

A normal gene may be A normal gene may be insertedinserted into the genome to into the genome to replace a nonfunctional gene. replace a nonfunctional gene.

An abnormal gene could be An abnormal gene could be swappedswapped for a normal gene for a normal gene through homologous recombination.through homologous recombination.

The abnormal gene could be The abnormal gene could be repairedrepaired through selective through selective reverse mutation.reverse mutation.

The The regulationregulation of a particular gene could be altered. of a particular gene could be altered.

STEPS OF GENE THERAPY

TYPES OF GENE THERAPY

GENE THERAPY

SOMATIC GENE THERAPY GERMLINE GENE THERAPY

Somatic gene therapy is further subdivided into – in vivo gene therapy ex vivo gene therapy

IN VIVO GENE THERAPY

In ‘in vivo’ gene therapy, genes are changed in cells present in the body.

EX VIVO GENE THERAPY

In ‘ex vivo’ gene therapy, cells are modified outside the body and then transplanted back in again.

The potential for discovering cures for terrible diseases Parkinson disease, Huntington disease, hemophilia, AIDS etc. leading to less pain and suffering. Enabling people to have children where natural conception is impossible. Increased availability of organs for transplant.

ADVANTAGES OF GENE THERAPY

DISADVANTAGES OF GENE THERAPY

Safety concerns - the risk Safety concerns - the risk of of detrimental genetic effectsdetrimental genetic effects (bad biological (bad biological consequences). consequences). Dangers associated with Dangers associated with after effectsafter effects of surgery such of surgery such as disability, blindness etc.as disability, blindness etc.

RECENT DEVELOPMENTS

In 2007, Moorfields Eye Hospital and In 2007, Moorfields Eye Hospital and London's Institute of Opthalmology London's Institute of Opthalmology announced the world's first gene therapy trial announced the world's first gene therapy trial for for inherited retinal diseaseinherited retinal disease..

In 2006, scientists at the National Institutes of Health (Bethesda, Maryland) have successfully treated metastatic melanoma in two patients using killer T cells genetically retargeted to attack the cancer cells. This study constitutes the first demonstration that gene therapy can be effective in treating cancer.

GENE THERAPY will go a long way in providing cure to many disabilities been summarized earlier. But one also has to think about the harm caused by it.