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GENE THERAPY
S O M AT I C C E L L T H E RA PY
E T H I C A L C O N S I D E RAT I O N S
D N A C A R R I E R F O R G E N E T H E RA PY

GENE
THERAPY
Gene therapy is the
transfer of genetic
material (DNA) for
the purpose of
treating human
diseases. Simply put,
a normal gene is
introduced into the
patient to replace an

GENE
Genetic diseases may be classified
THERAPY
into four categories
1.Single-gene changes
2.Multigene disorders
3.Mitochondrial disorders
4.Chromosome abnormalities

GENE
THERAPY

Single-gene changes
- single locus (gene) is defective and
responsible for the disease, is 100% heritable.
Examples include sickle cell anemia,
hyperchrolestrolemia, and cystic fibrosis
Multigene disorders
- two or more genes are defective and
responsible for the disease, is less than 100%
heritable. This disease may be dependent on

GENE
THERAPY

Mitochondrial disorders
- mutations in the mitochondrial DNA cause
diseases that affect many organ systems
Chromosome abnormalities
- sometimes complete chromosomes or parts
of the chromosome are missing, duplicated, or
modified in some way. Examples include Down
syndrome.

GENE
THERAPY

A gene that is inserted directly into a cell usually

does not function. Instead, a carrier called a
vector is genetically engineered to deliver the
gene. Certain viruses are often used as vectors
because they can deliver the new gene by
infecting the cell. The viruses are modified so
they can't cause disease when used in people.

GENE
THERAPY
Gene Therapy
has 2 types :
Somatic gene
therapy
Germline
gene therapy

SOMATIC CELL
InGENE
somatic cell
gene therapy, the
THERAPY

therapeutic genes are transferred

into any cell other than a gamete,
germ cell, gametocyte or
undifferentiated stem cell. Somatic
gene therapy represents
mainstream basic and clinical

SOMATIC CELL
There
are
three
routes
for
somatic
cell
GENE
THERAPY
gene therapy
1. Ex vivo cells are removed from
the body, incubated with the vector
and gene-engineered cells returned to
the body
2. In situ vector with the gene of

DNA CARRIERS FOR

GENE THERAPY

Crucial to gene therapy is the choice of the

vector. Vectors are either viruses or non-viruses.
The ideal vector for gene transfer must have the
following characteristics:
a) Can accomodate the gene of interest
b) Must be targeted to a limited cell type, must
not be immunogenic
c) Must be stable
d) Can easily be produced in high

VIRAL
Commonly
used
viral
vectors
are
VECTORS
retrovirus and adenovirus. Retroviruses
are RNA viruses that infect only
dividing cells and can insert into the
host genome randomly. Adenovirus
infects both dividing and nondividing
cells and does not integrate into the

NON-VIRAL
Non-viral
delivery
methods
may
be
VECTORS
safer than viral methods. Some of
these viral methods include:
a. Cationic liposomes
b. Naked plasmid DNA injection
A gene construct is made, composed of
a gene of interest with a promoter to

GENE
THERAPY
TODAY

Examples of gene therapy clinical trials for

diseases include:
SCID
Cystic Fibrosis
Advanced Central Nervous System (CNS)
Malignacy
Mesothelioma
Ornithine Transcarbamylase Deficiency
Hemophilia

SUCCESS OF
In 2003, the
first commercial gene
GENE
THERAPY
therapy was approved in China. The
therapy marketed under the name
Gendicine, treats head and neck
squamous cell carcinoma. This is an
injectable therapy that contains an
adenovirus vector with P53 tumor-

DEATH FROM
The first reported
case of death from
GENE
THERAPY

gene therapy was in 1999. Jesse

Gelsinger, 18 years old and was
suffering from ornithine
transcarbmalyse deficiency (OTC) that
causes a build up of ammonia, was
given the gene for this enzyme in an

QU
IZ