Gene therapy is a technique that involves correcting defective genes responsible for diseases. It can cure hereditary cancers and disorders like Parkinson's, Huntington's, hemophilia, and HIV/AIDS by altering a single gene. Gene therapy works by inserting a normal gene to replace a defective one, swapping an abnormal gene for a normal one, or repairing the abnormal gene. It offers potential cures for many terrible diseases but also carries safety risks like detrimental genetic effects and surgery-related disabilities. Recent developments include the first gene therapy trials for inherited retinal disease and genetically retargeted T cells treating cancer. While gene therapy may cure disabilities, its potential harm must also be considered.

1. Presented By: Disha Bedi
Class XII B
GENE THERAPY

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

3. 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.

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

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

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

7. WHAT IS GENE
THERAPY?
Gene therapy is a technique forGene therapy is a technique for
correctingcorrecting thethe defective genesdefective genes
responsible for developmentresponsible for development ofof
diseases.diseases.

8. GENE
 Genes, which areGenes, which are
carried oncarried on
chromosomeschromosomes, are the, are the
basic physical andbasic physical and
functional units offunctional units of
heredity. Whenheredity. When genesgenes
are alteredare altered so that theso that the
encoded proteins areencoded proteins are
unable to carry outunable to carry out
their normaltheir normal
functions,functions, geneticgenetic
disordersdisorders can result.can result.

9. WAYS TO GENE THERAPY
Researchers may use one ofResearchers may use one of several approachesseveral approaches forfor
correcting faulty genes:correcting faulty genes:
 A normal gene may beA normal gene may be insertedinserted into the genome tointo the genome to
replace a nonfunctional gene.replace a nonfunctional gene.
 An abnormal gene could beAn abnormal gene could be swappedswapped for a normal genefor a normal gene
through homologous recombination.through homologous recombination.
 The abnormal gene could beThe abnormal gene could be repairedrepaired through selectivethrough selective
reverse mutation.reverse mutation.
 TheThe regulationregulation of a particular gene could be altered.of a particular gene could be altered.

10. STEPS OF GENE THERAPY

11. 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

12. IN VIVO GENE THERAPY
In ‘in vivo’ gene
therapy, genes are
changed in cells
present in the body.

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

14. 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

15. DISADVANTAGES OF
GENE THERAPY
Safety concerns - the riskSafety concerns - the risk
ofof detrimental genetic effectsdetrimental genetic effects
(bad biological(bad biological
consequences).consequences).
Dangers associated withDangers associated with
after effectsafter effects of surgery suchof surgery such
as disability, blindness etc.as disability, blindness etc.

16. RECENT
DEVELOPMENTS
 In 2007, Moorfields Eye Hospital andIn 2007, Moorfields Eye Hospital and
London's Institute of OpthalmologyLondon's Institute of Opthalmology
announced the world's first gene therapy trialannounced the world's first gene therapy trial
forfor 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.

17. 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.