GENE

:Therapy
GENE
:The unit of heredity which is
transferred from parents to
offspring.
 What is a GENE?

Your body is Each cell has your Every

Your DNA are your Your GENES carry
made of CELL CHROMOSOME CHROMOSOME
GENES your traits.
S contains your DNA
What is a GENE MUTATION?
GENE THERAPY
A technique that uses•
GENE to treat, prevent
or cure a disease or
medical disorder.
GENE THERAPY
 Adding new copies of •a
GENE that is broken.
 Replacing a defective or
missing gene in a patient
cell with a healthy version
of that gene.
 Therapeutic
Genes Delivery Vehicle (Vector)

Human Genome

Healthy Body

Target Organ
 • GENE THERAPY•

In-Vivo Ex-
Introducing DNA
directly into cells
Vivo
Introducing normal
genes in isolated cells
while they are in the from patients.
patient.
• VECTORS
Vehicle of Gene
delivery.
• VECTORS in GENE
THERAPY•
• TYPES OF GENE THERAPY

SOMATIC
GENE THERAPY
GERMLINE
GENE THERAPY
 Genes is introduced in the
 Gene inserted into body
germ cells (egg & sperm
cells.
 Modification restricted to cell
 Modification will be
individual patient.
 Considered safe & ethically inherited by offspring.
 Not considered safe or
sound.
ethically sound.
GERMLINE GENE SOMATIC GENE
THERAPY THERAPY
 GENETIC
• ENGINEERING•
A process to alter the DNA makeup of an organism by
changing a single base pair (A-T, C-G), deleting region of
DNA or adding new segment of DNA.
• DISEASED PEOPLE CAUSED BY FIXING
THEIR GENES
(Clustered Regularly Interspaced
CAR-T CELL
CRISPR Short Palindromic Repeats) THERAPY
(Chimeric Antigen Receptor)
To treat sickle cell anemia. To treat Acute Lypmhocytic
Leukemia (ALL)

LUKTURNA SPINRAZA
FDA approved voretigene
FDA approved nusinersen to
neparvoveczyl to treat retinal
treat Spinal muscular atrophy.
dystrophy.
• ACTIVITY
Direction: Complete the table below. Research about gene/defect and clinical
features of the genetic disorder you pick.
GENETIC
GENE/ DEFECT
DISORDER
1. Hemophilia • When a blood vessel is injured,
special proteins in the blood called
“clotting factors” act to control
blood loss by plugging or patching
up the injury.
• People with this disorder have
lower than human levels of a
clotting factor.
CLINICAL FEATURES
• Easy bruising from an
early age
• Internal bleeding for no
obvious reason, especially
in the joints & muscles.
• Greater than normal
bleeding following injury
or surgery
• Abnormal heavy bleeding.

2.
What is the importance of Gene Therapy in
human health & society?

If you are suffering from a genetic disease

are you willing to undergo gene therapy?
Why?
• THANK
YOU! •