GENE THERAPY 2024.stud Ver

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GENE THERAPY
A. OVERVIEW
Genes contain DNA — the code that controls much of the body's form and function.
DNA controls everything from hair color and height to breathing, walking and
digesting food. Genes that don't work properly can cause disease. Sometimes these
genes are called mutations.
Gene therapy aims to fix a faulty gene or replace it with a healthy gene to try to cure
disease or make the body better able to fight disease. It holds promise as a treatment
for a wide range of diseases. Gene therapy is the use of genetic material to treat or
prevent disease.
The U.S. Food and Drug Administration (FDA) has approved gene therapy products
for several conditions, including cancer, spinal muscular atrophy, hemophilia and
sickle cell disease. But for most people, gene therapy is available only as part of a
clinical trial.
Clinical trials are research studies that help healthcare professionals find out whether
a gene therapy approach is safe for people. Clinical trials also help healthcare
professionals learn how gene therapy affects the body.
B. TERMINOLOGIES
Deoxyribonucleic acid (DNA) is the material that exists in every cell in your body
that holds your genetic code. It makes up your body’s instruction manual.
Genes are segments of DNA that determine our traits. Genes are the building blocks
for your body. Every human has between 20,000 and 25,000 different genes, half of
which are inherited from our biological mothers and the other half from our biological
fathers.
Chromosomes are long, bundled strands of DNA, each of which contains many
genes. In total, there are two sets of 23 chromosomes in a cell. Each set is inherited
from our biological parents.
DNA, genes and chromosomes work together to make you who you are.
Chromosomes carry DNA in cells. DNA is responsible for building and maintaining
your human structure. Genes are segments of your DNA, which give you physical
characteristics that make you unique. Together, your body has a complete
instruction manual that tells your cells how to behave.
Gene therapy seeks to alter genes to correct genetic defects and thus prevent or
cure genetic diseases.
Genetic engineering aims to modify the genes to enhance the capabilities of the
organism beyond what is normal.
C. TYPES OF GENE THERAPY

There are two types of gene therapy:
1. In vivo (inside your body): Scientists put the vector carrying the new genes
directly into your body via an injection or intravenous (IV) infusion.
2. Ex vivo (outside your body): Scientists extract cells from your body and
introduce them to the vector inside a petri dish. Then, your altered cells are
returned to your body, where they’ll hopefully multiply.
Each type has its own benefits:

Benefits of in vivo therapies Benefits of ex vivo therapies
can deliver vectors across the body, which is better at targeting specific
useful for bone or blood diseases organs and cell types
quicker and less invasive tend to pose fewer safety risks
D. DISEASES CURED BY GENE THERAPY

 Immunodeficiency disorders involve malfunction of the immune system,
resulting in infections that develop and recur more frequently, are more severe,
and last longer than usual.
 Congenital blindness is vision loss or severe vision impairment that is present

at birth. Not included in this category are other types of blindness that develop
because of injury or other medical conditions.
 Hemophilia is a rare disorder in which the blood doesn't clot in the typical way
because it doesn't have enough blood-clotting proteins (clotting factors).
 Sickle cell anemia or sickle cell disease affects the shape of red blood cells,
which carry oxygen to all parts of the body. Red blood cells are usually round
and flexible, so they move easily through blood vessels. In sickle cell anemia,
some red blood cells are shaped like sickles or crescent moons. These sickle
cells also become rigid and sticky, which can slow or block blood flow.
 Down syndrome is a genetic disorder caused when abnormal cell division

results in an extra full or partial copy of chromosome 21. This extra genetic
material causes the developmental changes and physical features of Down
syndrome.
E. BENEFITS OF GENE THERAPY:

 Fix genes that don't work properly. Faulty genes that cause disease could be
turned off so that they no longer promote disease. Or healthy genes that help
prevent disease could be turned on so that they could stop the disease.
 Replace genes that don't work properly. Some cells become diseased
because certain genes don't work properly or no longer work at all. Replacing
these genes with healthy genes may help treat certain diseases. For example, a
gene called p53 usually prevents tumor growth. Several types of cancer have
been linked to problems with the p53 gene. If healthcare professionals could
replace the faulty p53 gene, the healthy gene might cause the cancer cells to
die.
 Make the immune system aware of diseased cells. In some cases, your
immune system doesn't attack diseased cells because it doesn't see them as
intruders. Healthcare professionals could use gene therapy to train your immune
system to see these cells as a threat.
F. RISKS OF GENE THERAPY:

This technique presents risks, including:
 Unwanted immune system reaction. Your body's immune system may see the
newly introduced viruses as intruders. As a result, it may attack them. This may
cause a reaction that ranges from swelling to organ failure.
 Targeting the wrong cells. Viruses can affect more than one type of cell. So it's
possible that the changed viruses may get into cells beyond those that aren't
working properly. The risk of damage to healthy cells depends on which type of
gene therapy is used and what it's used for.
 Infection caused by the virus. It's possible that once the viruses get into the
body, they may once again be able to cause disease.
 Possibility of causing errors in your genes. These errors can lead to cancer.
G. WHAT ARE THE ETHICAL ISSUES SURROUNDING GENE THERAPY?

Because gene therapy involves making changes to the body’s basic building blocks
(DNA), it raises many unique ethical concerns. The ethical questions surrounding
gene therapy and genome editing include:
1. How can “good” and “bad” uses of these technologies 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?
4. Could the widespread use of gene therapy make society less accepting of
people who are different?
5. Should people be allowed to use gene therapy to enhance basic human traits
such as height, intelligence, or athletic ability?
Current research on gene therapy treatment has focused on targeting body (somatic)
cells such as bone marrow or blood cells. This type of genetic alteration cannot be
passed to a person’s children. Gene therapy could be targeted to egg and sperm
cells (germ cells), however, which would allow the genetic changes to be passed to
future generations. This approach is known as germline gene therapy.
The idea of these germline alterations is controversial. While it could spare future
generations in a family from having a particular genetic disorder, it might affect the
development of a fetus in unexpected ways or have long-term side effects that are
not yet known. Because people who would be affected by germline gene therapy are
not yet born, they can’t choose whether to have the treatment. Because of these
ethical concerns, the U.S. Government does not allow federal funds to be used for
research on germline gene therapy in people.
References:
https://medlineplus.gov/genetics/understanding/therapy/ethics/
https://my.clevelandclinic.org/health/treatments/17984-gene-therapy
https://www.mayoclinic.org/tests-procedures/gene-therapy/about/pac-20384619#:~:text=Gene
%20therapy%20aims%20to%20fix,%2C%20diabetes%2C%20hemophilia%20and%20AIDS.
https://patienteducation.asgct.org/gene-therapy-101/gene-therapy-basics
https://my.clevelandclinic.org/health/body/23064-dna-genes--chromosomes

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SOC.

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C. TYPES OF GENE THERAPY

Each type has its own benefits:

D. DISEASES CURED BY GENE THERAPY

 Congenital blindness is vision loss or severe vision impairment that is present

 Down syndrome is a genetic disorder caused when abnormal cell division

E. BENEFITS OF GENE THERAPY:

F. RISKS OF GENE THERAPY:

G. WHAT ARE THE ETHICAL ISSUES SURROUNDING GENE THERAPY?

1. How can “good” and “bad” uses of these technologies be distinguished?

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