GENE THERAPY
GROUP 5
AROMIN, CHARLES
VINCENT G.
BALANZA, MA. JENICE G.
BLANCO, JOHN AARON T.
JADORMEO, ELISHA GWYN
Gene Therapy: Terms to Know
- Capsid Protein shell surrounding viral DNA that helps target
the genetic material to specific cell types
- Cell The smallest structural and functional unit of an organism
- DNA A molecule that carries genetic instructions
- Gene cassette A small piece of DNA containing the therapeutic
gene and instructions for the cell on how to use the gene
- Nucleus The central part of a cell; contains the DNA used to
make proteins
- Protein Essential structural and functional components of all
living organisms, e.g. body tissues (muscle, hair, etc.), enzymes
and antibodies
- Vector May refer to the DNA molecule itself or to the carrier
construct containing the DNA to be delivered to a cell.
GENES
WHAT IS A GENE AND HOW DOES IT WORK?
• In the center of every cell in your body is an area
called the nucleus. The nucleus contains your
DNA (deoxyribose nucleic acid), which is the
genetic code you inherited from each of your
parents.
• Genes are made up of anywhere from a few
hundred to a few million pieces of DNA. You have
46 chromosomes, which are each made up of
thousands of genes. Each person has between
20,000 and 25,000 genes in their body. You have
2 copies of every gene, 1 inherited from your
mother and 1 from your father.
• Genes contain instructions for the cell on
how to make proteins. Proteins are
necessary to perform most life functions.
Genes tell cells how to work, control our
growth and development, and determine
what we look like and how our bodies
work. They also play a role in the repair
of damaged cells and tissues.
GENE
THERAPY
-First discovered in the middle of
the 1970’s researchers were able
to isolate certain genes from DNA.
During the 1980’s the term gene
therapy came about and propelled
research further.
- Gene therapy is a treatment in
which corrects errors in a person’s
genetic code.
- It has been approved for
commercial use in Europe for the
first time (James Gallagher, 2012).
- Gene therapy is a technique that uses
genetic material (a piece of DNA) for
the long-term treatment of genetic
disorders. This may involve delivering a
copy of a healthy or therapeutic gene,
repairing a faulty gene, and/or altering
the degree to which a gene is turned
‘on’ and ‘off’.
HOW DOES
GENE THERAPY
WORK?
BASIC STEPS OF GENE THERAPY
• The faulty gene that causes a specific
condition must be identified.
• The location of the affected cells in the
body’s tissues or organs must be
pinpointed.
• A working version of the gene must be
available.
• The working version of the gene has to
be delivered to the cell
TYPES OF GENE THERAPY
TREATMENT
SOMATIC GENE THERAPY
- Replaced, working gene that is inserted into
the cells in the body that are affected would
cure the individual. It would not prevent their
children from inheriting the original faulty
gene, however, as these are carried on the
sperm and egg cells (called ‘germ’ cells).
GERMLINE GENE THERAPY
- It involves modifying the genes in the egg or
sperm cells, which will then pass any genetic
changes to future generation.
TYPES OF SOMATIC GENE THERAPY
IN VIVO IN VIVO
• Direct delivery of
therapeutic gene into
target cell into patients
body.
• It can be the only possible
options in patients where Genes are
individual cells cannot be transferred into
cultured in vitro in cells while still in
sufficient numbers (e.g. the patient
brain cells).
TYPES OF SOMATIC GENE THERAPY
EX VIVO EX VIVO
• A procedure that entail
Cells are taken the extraction of cells
from the patient
from the patient's body
(for instance bone-
marrow cells), genetic
Gene is modification of the cells
modified in using appropriate vectors
the lab
or other DNA-transfer
methods and
reimplantation of the
Cells are transferred
back into the patient cells in the patient.
ADA ( Adenosine demeans deficiency)
- also known as ADA Deficiency, is the first
disease that was approved for gene therapy
.
- A normal ADA gene should produce an
enzyme that is called adenosine deaminase
which a person with ADA deficiency does
not produce. People with this deadly
disease are seriously prone to the most
minor of illnesses and If untreated, life may
only take a couple of years and
replacement ADA is limited.
CONS
- After initially receiving a type of gene therapy, the
patient's immune system may react to the foreign
vector, causing fever, severe chills (called rigors),
drop in blood pressure, nausea, vomiting, and
headache.
- Damage healthy cells
- Can affect the future generations.
- It may target the wrong cells.
- Infection caused by the virus.
- Possibility of causing a tumor.
PROS
-Replacing mutated genes
-Fixing mutated genes
-Making diseased cells more evident to the
immune system
- Can control or eliminate hereditary
diseases.
BIOETHICS
Somatic gene therapy has had some successes but still faces
significant technical challenges and it can pose significant risks
to patients and some have died in experiments.
Germline gene therapy it involves deliberate, inheritable
changes in the genome that poses risks not only to patients but
also to future GENERATIONS related to tampering with human
nature, enhancing human traits, parental control over children,
discrimination, social justice and eugenics.
The distinction between therapy (which aims to prevent or treat
disease) and enhancement (which aims to improve human traits)
should be distinguished because the concept of disease is not
purely objective and includes social, cultural and ethical
dimensions.
ETHICAL CONSIDERATION
- The first issue targets putting human fate in our own
hands giving us the ability to manufacture future
generations.
- It would be used to create a superior race.
- Considered the religion.
- Invasion of privacy is also concern.
- Consider human dignity.
- Economic difficulties, particularly with regard to wealth
distribution.
- Political and cultural conflicts, as well as the scarcity of
studies evaluating the impacts of the use of gene therapy
on human .
- Cause discrimination to families with genetic diseases.
• Investigators and institutions should take
appropriate steps to protect the rights and welfare
of gene therapy patients participating in clinical
research.
• In thinking about the balance of benefit and risks
of a gene therapy study, it is important to consider
the safety and efficacy of the proposed research
and the availability of alternatives
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