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M12: Learning Activity: Guide Questions: 1. How Gene Therapy or Cell Therapy Can Help Cure Diseases?

Gene and cell therapy can help cure diseases by replacing defective genes, adding genes to help the body fight disease, or modifying cells outside the body before reintroducing them. These therapies treat genetic disorders caused by single gene defects as well as some cancers and infectious diseases. However, there are risks such as long-term effects being unknown and no guarantees of a cure, as well as ethical issues involving embryonic stem cell research and destruction of embryos.

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Kimberly Wealth Meonada Magnaye
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69 views4 pages

M12: Learning Activity: Guide Questions: 1. How Gene Therapy or Cell Therapy Can Help Cure Diseases?

Gene and cell therapy can help cure diseases by replacing defective genes, adding genes to help the body fight disease, or modifying cells outside the body before reintroducing them. These therapies treat genetic disorders caused by single gene defects as well as some cancers and infectious diseases. However, there are risks such as long-term effects being unknown and no guarantees of a cure, as well as ethical issues involving embryonic stem cell research and destruction of embryos.

Uploaded by

Kimberly Wealth Meonada Magnaye
Copyright
© © All Rights Reserved
We take content rights seriously. If you suspect this is your content, claim it here.
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Download as DOCX, PDF, TXT or read online on Scribd
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M12: Learning Activity

Write your analysis essay regarding gene and cell therapy.

Guide Questions:
1. How gene therapy or cell therapy can help cure diseases?

Gene therapy can be used to reduce levels of a disease-causing version of a


protein, increase production of disease-fighting proteins, or to produce new/modified
proteins. A cell therapy is the transfer of intact, live cells into a patient to help lessen or
cure a disease.

In gene therapy, scientists can do one of several things depending on the


problem that is present. They can replace a gene that causes a medical problem with
one that doesn’t, add genes to help the body to fight or treat disease, or turn off genes
that are causing problems. In order to insert new genes directly into cells, scientists use
a vehicle called a “vector” which is genetically engineered to deliver the gene. Viruses,
for example, have a natural ability to deliver genetic material into cells, and therefore,
can be used as vectors. Before a virus can be used to carry therapeutic genes into
human cells, however, it is modified to remove its ability to cause an infectious disease.
Gene therapy can be used to modify cells inside or outside the body. When it’s done
inside the body, a doctor will inject the vector carrying the gene directly into the part of
the body that has defective cells. In gene therapy that is used to modify cells outside of
the body, blood, bone marrow, or another tissue can be taken from a patient, and
specific types of cells can be separated out in the lab. The vector containing the desired
gene is introduced into these cells. The cells are left, to multiply in the laboratory, and
are then injected back into the patient, where they continue to multiply and eventually
produce the desired effect.

2. What kinds of diseases do gene and cell therapy treat?

Diseases treated by gene therapy. Gene Therapy was initially meant to introduce
genes straight into human cells, focusing on diseases caused by single-gene defects,
such as cystic fibrosis, hemophilia, muscular dystrophy, and sickle cell anemia. Three
types of diseases for gene therapy can be distinguished. First Disease is monogenic
disorders, single locus (gene) is defective and responsible for the disease, 100%
heritable. For example, Sickle cell anemia, Severe Combined Immunodeficiency, Cystic
fibrosis, Hemophilia, Duchenne muscular dystrophy, Huntington’s disease, Parkinson’s,
Hypercholesterolemia, Alpha-1 antitrypsin, Chronic granulomatous disease, Fanconi
Anemia and Gaucher Disease. Second Disease is Polygenic disorders, multiple genes
involved, disease may be dependent on environmental factors and lifestyle. Examples:
Heart disease, Cancer, Diabetes, Schizophrenia and Alzheimer’s disease. Last is
Infectious diseases, such as HIV.

3. What is the difference between gene therapy and cell therapy?

In the broadest sense, gene therapy is the introduction, removal, or change in


the content of a person’s genetic code with the goal of treating or curing a disease. The
transferred genetic material changes how a single protein or group of proteins is
produced by the cell. Gene therapy can be used to reduce levels of a disease-causing
version of a protein, increase production of disease-fighting proteins, or to produce
new/modified proteins. Gene therapy involves the transfer of genetic material, usually in
a carrier or vector, and the uptake of the gene into the appropriate cells of the body.
While cell therapy involves the transfer of cells with the relevant function into the
patient. Some protocols utilize both gene therapy and cell therapy. Cell therapy is the
transfer of intact, live cells into a patient to help lessen or cure a disease. The cells may
originate from the patient (autologous cells) or a donor (allogeneic cells). The cells used
in cell therapy can be classified by their potential to transform into different cell types.

4. What are the different approaches to gene and cell therapy?

Gene Therapy is the use of genetic material in the treatment or prevention of


disease. Typically, genetic material, such as a working copy of a gene, is delivered to
cells using a vector. A vector is often derived from a virus. For safety, all viral genes are
removed, and the vector is modified to only deliver therapeutic genes into the cells.
Once in the cell, a working copy of the gene will help make proteins despite the
presence of a faulty gene. Achieving the normal expression and function of proteins
makes a big impact on our overall health. While Cell Therapy is the transfer of cells into
a patient with the goal of improving a disease. Some cell therapies are routine, like blood
transfusions. One approach is gene-modified cell therapy, which removes the cells from
the patient’s body, then a new gene can be introduced, or a faulty gene can be
corrected. The modified cells are then put back into the body. An example of this
approach is CAR-T cell therapy.

5. What risks are associated with gene and cell therapy?

First, it cannot be given another dose. In the event a person is not satisfied with
the outcome, the person cannot receive another dose of the gene therapy. Participating
in a clinical trial may also prevent future participation in other trials or from receiving
other types of treatments. Second, long-term effects unknown. Gene therapy can be an
alteration for the lifetime, so people should be aware that there could be long term
effects (both good or bad) that are unknown at this time. Third, informed consent.
Before participating in a clinical trial, a member of the research team should review any
potential risks and benefits with the patient or caregiver. Fourth, No guarantees.
Therapies being studied in clinical trials are not a guaranteed cure and cannot guarantee
beneficial results. There is always a chance that the investigational treatment may not
work.

6. What are the ethical issues associated with gene and cell therapy?

Ethical issues in genetic therapy involve the use of genetic science for human
good and the avoidance of preventable harms. Immediate issues are the control and
confidentiality of results of genetic testing, possible discrimination against those found to
have genetic diseases, and the just allocation of beneficial genetic therapies. One of the
ethical issues raised by research using cell therapy is the development of embryonic
stem cells from fertilized eggs. Because obtaining embryonic stem cells requires the
destruction of human embryos, it runs up against existing ethical taboos. Some religious
and anti-abortion groups argue that the destruction of embryos after stem cells is
tantamount to the death of a life. In addition, in order to avoid "human fertilized eggs
research is killing life", some people use human and animal cell fusion experiment.
Whatever the outcome is, it is now highly controversial and worth pondering.

7. What are stem cells? Why are stem cells so important in gene and cell therapy?

Stem cells are cells that can self-renew and can mature into at least one type of
specialized cell. Stem cells provide two major benefits for gene and cell therapy. First,
they provide a cell type that can self-renew and may survive the lifetime of the patient.
Second, stem cells provide daughter cells that mature into the specialized cells of each
tissue. These differentiated daughter cells can replace the diseased cells of the afflicted
tissue(s). Therefore, gene and cell therapy that uses stem cells theoretically improves
the disease condition for as long as those modified stem cells live, potentially the lifetime
of the patient.

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