Gene Therapy
Gene Therapy
Gene Therapy
THERAP BSA-2A
FABIAN, MARK FRANCIS
Y ESPINO, MARIELA
SAET, MICHAELLA VANEIREEN
YUMUL, PAULA
Medical science has detected
many human diseases related to
defective genes. These types of
diseases are not curable by
traditional methods like taking
readily available medicines.
Gene therapy is a
potential method to
either treat or cure
genetic-related
human illnesses.
G
GEEN
NEE T
THHE
ERRA
A PP Y
Y
-is an experimental technique
that uses genes to treat or
prevent disease. In the future,
this technique may allow
3 to treat a disorder by
doctors
inserting a gene into a patient's
cells instead of using drugs or
surgery.
In 2015, a team of researchers at the
Harvard Medical School and the Boston
Children's Hospital stated that they were
able to restore basic hearing in
genetically deaf mice using gene
therapy.
The Boston Children's Hospital
research team also reported that they
have restored a higher level of
hearing—down to 25 decibels which
is actually equivalent to a whisper.
They used an improved gene therapy
vector developed at the Massachusetts
Eye and Ear that was identified as
“Anc80" which enables the transfer of
genes to the inaccessible outer hair cells
when introduced into the cochlea
(Fliesler, 2017).
HUMA
It was first
N Viewed as
realized in GENE insertion
1971 when the foreign DNA
first
recombinant
THERA into a
patient's tissue
DNA PY that eradicates
experiments the targeted
were planned. disease.
Human
Human gene
gene
therapy
therapy
Inspired by the success of
recombinant DNA technology which
occurred over the last 20 years.
THE
BASIC
PROCESS
There are several
approaches to gene
therapy. These are the
following (Fliesler,
2017):
THE
THE BASIC
BASIC
PROCESS
PROCESS
Replacement of mutated gene that
causes disease with a healthy copy of
the gene.
THE
THE BASIC
BASIC
PROCESS
PROCESS
Inactivation of a mutated gene
that is functioning improperly.
THE
THE BASIC
BASIC
PROCESS
PROCESS
Introducing a new gene into the
body to help fight a disease.
A gene cannot be
directly inserted into
a human gene or cell.
A gene is inserted
into another gene
using a carrier or
vector.
At present, the most common
type of vectors are viruses that
have been genetically changed to
carry normal human DNA.
Viruses have evolved
a way of
encapsulating and
transporting their
genes to human cells
in a pathogenic
manner (Science
TWO TYPES OF
GENE THERAPY
Somatic gene
therapy
Somatic gene therapy involves the
manipulation of genes in cells that will be
helpful to the patient but not inherited to
the next generation (Nimsergern, 1988).
Germ-line
gene
therapy
Germ-line gene therapy involves the
genetic modification of germ cells or the
origin cells that will pass the change on to
the next generation (Your Genome, 2017).
STEM CELL
GENE
THERAPY
STEM
CELLS
• are mother cells that have the
potential to become any type of
cell in the body
STEM
CELLS
• can become cells of the blood,
heart, bones, skin, muscles, brain,
among others.
STEM
• StemCELLS
cells are derived from
different sources. Two of which
are embryonic and somatic stem
cells.
EMBRYONIC
STEM
CELLS
The embryos are usually extras that have
been created in IVF (in vitro fertilization)
clinics where several eggs are fertilized in a
test tube then implanted into a woman
(Crosta, 2013).
SOMATIC
STEM
CELLS
• are cells that exist throughout the body
after embryonic development and are
found inside of different types of tissue.
SOMATIC
STEM
CELLS
It is generally thought that adult or somatic stem
cells are limited in their ability to differentiate
based on their tissue of origin, but there is some
evidence to suggest that they can differentiate to
become other cell types (Crosta, 2013).
THE
B I O E TH I CS
O F G EN E
T H E
ER RAPY
APY
There are ethical issues involved in
gene therapy. Some of the inquiries
cited are (Genetics Home Reference,
2017):
1. How can "good" and "bad" uses
of gene therapy be distinguished?
2. Who decides which traits are
normal and which constitute a
disability or disorder?
3. Will the high costs of gene therapy
make it available only to the
wealthy?