Gene

Therapy
Genetics and Cell Biology
LEARNING OBJECTIVES
1. Analyze the various methods and
techniques used in gene therapy and,

2. Relate the importance of genetic therapy

towards genetic diseases and,
ACTIVITY!
Genetic Engineering
Biotechnology
Mutations
Gene Therapy
 CONTENTS
TYPES OF GENE APPROACHES OF
1 2
THERAPY GENE THERAPY

BENEFITS AND
3 PROCESS 4
How gene therapy is done? POSSIBLE RISKS
What is Gene
Therapy?
Gene Therapy
is a technique that uses a
gene(s) to treat, prevent or cure
a disease or genetic disorder.
 Inherited Genetic Disorders
Hemophilia Sickle Cell Disease
 Acquired Disorders

Leukemia
1
TYPES OF GENE
THERAPY
Depending on which types
of cells are treated
 TYPES OF GENE
THERAPY

SOMATIC GERMLINE
• Targets body tissues • Targets germinal or
• Not heritable reproductive cells
• Inheritable
TYPES OF GENE
THERAPY
2
APPROACHES OF
GENE THERAPY
 APPROACHES OF GENE
THERAPY
a) In-vivo gene therapy - the target cell is
fixed in the body of the living organism
and the therapeutic gene or foreign gene
is inserted into the target cell through a
vector.
b) Ex-vivo gene therapy - the cells are
taken out from the body and grown in
the laboratory.
APPROACHES OF GENE
THERAPY
Transfer of a new gene
into the nucleus of a cell
via a viral vector.
 DIFFERENT GENE THERAPY
APPROACHES USE IN
TREATMENT
1. Gene addition- inserts a new copy of a gene into your
cells.
2. Gene silencing- transfers genetic material that
prevents the activity of a gene that’s already in a cell.
3. Gene editing- Gene editing modifies parts of your
DNA by altering or deleting elements within your gene.
3
HOW GENE
THERAPY IS
DONE?
HOW GENE THERAPY IS DONE?

REPLACING FIXING
MAKING DISEASE
MUTATED MUTATED
CELLS MORE
GENES GENES
EVIDENT TO THE
IMMUNE SYSTEM
GROUP
ACTVIT
Y
4
BENEFITS AND
POSSIBLE RISK
BENEFITS
a) Offers Hope
b) Give Long-lasting and Timeless
Effect
c) Treat Genetic Disorders
d) Aren’t limited to Humans only
 POSSIBLE
RISKS
This technique presents the following risks:

a. Unwanted immune system reaction.

b. Targeting the wrong cells.
c. Infection caused by the virus.
THANKS FOR
LISTENING!