GENE THERAPY

Presented by:
MD KAMAL HOSSAIN 12101073
MD. AMIT HASAN
16304010
HASAN TAWQIR AHMED 14101203
TANVIR REZWAN 13304021

WHAT IS GENE THERAPY ?

Definiton: 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 a healthy copy of the gene.
Inactivating, or knocking out, a mutated
gene that is functioning improperly.
Introducing a new gene into the body to
help fight a disease.

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.
Also raises controversy:
Some people view this type of therapy as
unnatural, and liken it to "playing God.
Others have concerns about the technical
aspects.

SOMATIC 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.
Transporting the gene to the target cells or
tissue is also problematic.
Appropriate and acceptable for many
disorders, including cystic fibrosis, muscular
dystrophy, cancer, and certain infectious
diseases.

Types of somatic gene

therapy

BASIC PROCESS OF
GENE THERAPY

VIRAL VECTOR
NON VIRAL VECTOR

1.
2.

The two major classes of methods :

recombinant viruses VIRAL VECTOR
naked DNA or DNA complexes NONVIRAL VECTOR

VIRAL VECTOR

Virus bind to their hosts and introduce their genetic

material into the host cell.

Plausible strategy for gene therapy, by removing the

viral DNA and using the virus as a vehicle to deliver
the therapeutic DNA.

The viruses used are altered to make them safe,

although some risks still exist with gene therapy.

adeno-associated viruses, lentiviruses, pox viruses,

alphaviruses, and herpes viruses.
Delivery of genes by a virus is termed transduction
and the infected cells are described as transduced.

NON VIRAL VECTOR

Researchers are studying gene therapy

for a number of diseases, such as
Severe combined immunodeficiencies (SCID)
Haemophilia
Parkinson's disease
Cancer
HIV

Success of Gene Therapy

Retinal disease
Color blindness
Adrenoleukodystrophy

ADVANTAGES OF GENE
THERAPY
Give a chance of a normal life to
baby born with genetic disease.
Give hope of healthy life to cancer
patient.
For certain disease that do not
have any cure except gene
therapy, it could save many lives

DISADVANTAGES OF GENE
THERAPY

The genetic testing, screening and research in

finding the availability of certain gene is very
controversy.
May increase rate of abortion if prenatal test
regarding baby with genetic disease is done.
The cost is very high.
Cosmetic industry may monopolized this gene
therapy if it is used in enhancing beauty and in
vanishing the aging effect, rather than used
for treatment of a disease.